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Regulatory Alert | Aspen Xchange

Regulatory Alert

The US FDA is actively monitoring and assessing how current vaccines, therapeutics, and diagnostics are impacted by emerging COVID-19 variants and will issue guidance for ongoing medical product development against these variants. In a recent statement, the FDA's acting commissioner Janet Woodcock, MD, said the agency has been monitoring potential new coronavirus variants since the start of the COVID-19 pandemic, and has been actively working with medical product sponsors and international partners to evaluate the impact new COVID-19 variants have on authorized medical products. The agency is continuing to take an “all-hands-on-deck” approach to the pandemic, she said. “As the public health agency responsible for regulating medical products, we must ensure that health care providers have the most up-to-date diagnostics, treatments and vaccines in their toolbox to fight this pandemic.”
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A new collaborative initiative is bringing the perspective of non-European Union regulators into the evaluation process for vaccines and therapeutics to combat COVID-19. The pilot program, dubbed OPEN, debuted in December 2020. This week, the EMA published a series of questions and answers fleshing out how the collaboration is working. The extra-EU regulators are contributing to EMA’s COVID-19 pandemic task force, as well its human medicines committee (CHMP); OPEN pilot participation is ordinarily capped at two outside members per authority, though exceptions could be made, said EMA.
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The EMA this week released draft versions of the third revision to its good pharmacovigilance practice (GVP) module XVI guideline on risk minimization measures and its second addendum to the guideline on methods for evaluating the effectiveness of risk minimization measures for public consultation. The deadline to comment on the documents is April 28th 2021 and the EMA says it expects the revised guideline and new addendum to come into effect in late 2021 or early 2022. The current versions of the guideline came into effect since March 2017. Revisions to the 30-page GVP guideline include changes to several sections, the addition of three new sections and the deletion of an appendix on survey methodologies, the latter of which has been integrated into Addendum II of the module.
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The US FDA's Center for Biologics Evaluation and Research (CBER) has released its list of draft and final guidances it plans to issue in 2021. The list contains just 14 guidances, half of which will be draft documents, down from 31 guidances in 2020 and 20 guidances in 2019. Of those 14 guidances, only a few are new additions to the list, with the remainder being carried over from the previous year. CBER was able to cross more than 20 guidances off its list in 2020, despite taking on the development and issuance of five guidances related to COVID-19, including guidance on the development and licensure and emergency use authorization of COVID-19 vaccines.  As was the case last year, CBER says it is not “bound by this list of topics, nor required to issue every guidance document on this list,” and that it is not precluded from issuing guidance beyond the topics that appear on the list.
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In a nearly 350-page report to Congress on the federal government’s COVID-19 response, the Government Accountability Office (GAO) calls on the US FDA to review its inspections approach and come up with a plan to address its looming backlog. In the early days of the pandemic, the FDA halted non-mission-critical foreign and domestic inspections and relied on alternative tools, such as inspection reports from foreign regulators, records requests and product sampling, to complement its oversight activities. Unsurprisingly, the pandemic has taken its toll on FDA’s inspections program, cutting the number of drug establishment inspections the agency carried out in FY2020 to less than half of what it had done in the previous two fiscal years. 
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The U.S. FDA has extended the review period for Biogen and Eisai’s BLA for aducanumab for Alzheimer’s disease. The drug has been fraught with controversy—and unexpected hopes. The decision by the FDA on the drug application was expected on March 7, but is now delayed until June 7, 2021. In March 2019, Biogen and Eisai announced they were discontinuing the global Phase III clinical trials ENGAGE and EMERGE of aducanumab in patients with mild cognitive impairment for Alzheimer’s and mild Alzheimer’s dementia. An independent data monitoring committee indicated they were unlikely to hit their primary endpoint. But in October 2019, the two companies announced plans to pursue regulatory approval for the drug. It turned out, the Phase III EMERGE trial met its primary endpoint, showing a significant decrease in clinical decline.
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In an update to its guidance on conducting clinical trials amid the COVID-19 pandemic, the US FDA this week explained COVID-19 treatments and vaccines used under an emergency use authorization (EUA) are not considered investigational products for the purposes of clinical trial exclusion criteria. The guidance, first issued in March 2020, has been updated several times throughout the pandemic to address questions raised by industry about the conduct of clinical trials during the pandemic. Many clinical trial protocols feature exclusion criteria that screens out participants who have received another investigational medical product. This has led to some confusion among clinical trial sponsors.
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The US FDA's Center for Drug Evaluation and Research (CDER) has released a list of the new and revised draft guidances it plans to release in 2021. The agenda, while oftentimes aspirational, provides insights as to what the agency’s drug center’s new guidance priorities are for the coming year. However, the list does not include draft or revised guidances the agency plans to finalize in 2021. Many of the planned guidances on the list were included in previous years’ agendas, such as the proposed clinical pharmacology draft guidance “Assessing the Effects of Food on Drugs in INDs or NDAs – General Considerations”, and updated guidance on “Pharmacogenomic Data Submission” and “Pharmacokinetics in Patients with Impaired Renal Function – Study Design, Data Analysis and Impact on Dosing and Labeling”. In total, there are 105 guidances included on the agenda, 42 of which are new for 2021. 
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During the pandemic, the US FDA continued to attempt to meet goals mandated by its various user fee programs – and, generally, the agency succeeded, though a steep drop in inspections hampered some efforts. The details of how well FDA’s Center for Drug Evaluation and Research (CDER) met its user fee commitments were laid out in a January 26th report covering the third and fourth quarters of federal fiscal year 2020 (FY20), which ended September 30th, 2020. Across all approval types, the number of cases where pre-approval inspections were skipped in favor of alternative approaches rose from 48% in the third quarter to 60% by the fourth quarter of FY20.
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Effective use of real-world data and other data gathered outside the clinical trial arena requires a unified, big-picture approach, according to a new report from the first-ever big data stakeholder forum held by the EMA. The regulator’s report on the December 15th, 2020 forum highlighted the importance of collaboration in addressing some of the challenges of bringing real-world data (RWD) into the drug development process. Some of these include data quality, the need for robust study methods, and “establishing the evidentiary value and regulatory accessibility of evidence generated.”
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Federal criminal charges have been filed against a man previously warned by the FDA for peddling unproven and unauthorized “vaccines” against COVID-19. Johnny Stine, president of Oregon-based North Coast Biologics, previously received a May 2020 warning letter jointly issued by the US FDA and the FTC. In the letter, the agencies ordered him to cease “misrepresentations” made on Facebook and LinkedIn, as well as the firm’s website. That May warning letter was preceded by a April 23rd “cease and desist” letter from Washington’s Attorney General, Bob Ferguson. Noting that he had directed an investigator to review Facebook posts made by Stine, Ferguson wrote that the investigator found that Stine was “making false or unsupported claims about your so-called “vaccine” that may mislead consumers during the public health emergency related to the COVID-19 pandemic.”
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Despite the pandemic, the European Medicines Agency (EMA) issued many more positive recommendations for human medicines in 2020 than in the previous year, according to a new report from the agency highlighting the year’s regulatory activities. In 2020, 97 new medicines were recommended for authorization by EMA; of these, 39 contain a new active substance (NAS). In 2019, EMA issued 66 positive opinions for medicines, 30 of which contained a NAS. The total numbers of new recommendations include conditional marketing authorizations such as the ones issued for the COVID-19 treatment Veklury (remdesivir) and Comirnaty, the COVID-19 vaccine co-developed by Pfizer and BioNTech.
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Manufacturers of cell and gene therapies have a new guidance from the US FDA that provides pandemic-related manufacturing considerations. The guidance specifically addresses both licensed and investigational cell and gene therapy (CGT) manufacture, and “is intended to supplement the recommendations to drug and biological product manufacturers provided in FDA’s ‘Good Manufacturing Practice Considerations for Responding to COVID-19 Infection in Employees in Drug and Biological Products Manufacturing; Guidance for Industry’ issued in June 2020,” according to the guidance. Industry-specific issues addressed in the guidance include cells and tissues recovered from donors and the particular methods by which a CGT product will be manufactured, such as cell expansion, viral reduction steps, and formulation. 
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Janet Woodcock, who has led the FDA’s drug review efforts for years, is slated to oversee the agency on an interim basis after current Commissioner Stephen Hahn leaves latere today. President-elect Joe Biden has yet to nominate an FDA commissioner. Dr. Hahn has designated Dr. Woodcock as interim commissioner at least until a permanent commissioner is nominated and confirmed, a designation that is expected to be seconded Wednesday by the Biden transition team. Dr. Woodcock joined the FDA in 1986, and has most recently overseen the FDA’s Center for Drug Evaluation and Research. The Biden transition team didn’t immediately respond to a request for comment on the FDA post, and Dr. Woodcock declined to comment.
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Pfizer and BioNTech plan to seek full regulatory approval, or licensure, for their coronavirus vaccine in the first half of the year. As of now, Pfizer-BioNTech’s jabs, and another vaccine developed by Moderna, were rolled out in the U.S. under the Food and Drug Administration’s emergency use approval (EUA), or authorization intended to address serious, threatening diseases where there are no other alternatives. Full FDA approval, however, requires a so-called Biologics License Application, or BLA. "Pfizer and BioNTech are planning to submit a Biologics License Application (BLA) during the first half of 2021 to secure full regulatory approval for BNT162b2," reads a statement emailed to Fox News. "Once filed, we expect the FDA to make a final decision in the second half of 2021."
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Documents related to COVID-19 vaccines that were unlawfully accessed in a cyberattack against the European Medicines Agency (EMA) and subsequently leaked online were intentionally manipulated, the agency says. The EMA first disclosed the hack in December and said at the time it had launched a full investigation in cooperation with law enforcement and other authorities. Since then, vaccine frontrunners Pfizer and BioNTech and Moderna have released statements acknowledging that documents related to their COVID-19 vaccine submissions were accessed. Now, in its latest update, the EMA has disclosed that the leaked documents included internal confidential email correspondence from November relating to its evaluation processes for COVID-19 vaccines. “Some of the correspondence has been manipulated by the perpetrators prior to publication in a way which could undermine trust in vaccines."
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Most novel drugs approved by the US FDA in 2020 made their way through the approvals process using one or more expedited approval pathways, and over half of sponsors received the incentives that accompany orphan drug designation, according to an FDA report detailing the year’s approvals. Novel drug approvals continued at a brisk clip, averaging over one per week in 2020. Despite the pandemic the 53 novel drugs approved in 2020 bested 2019’s 48 approvals but fell short of the 59 approved in 2018. In a report on the FDA’s novel drug approvals for 2020, FDA noted that 40%, or 21 of 53 approved drugs, are considered first-in-class. Rare diseases were addressed by 31 of the novel drugs, or 58%.
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Discussions continue between industry and the US FDA regarding the upcoming reauthorization of user fees for prescription drugs, biologics and generic drugs. After public hearing and a first round of meetings that gave a peek into priorities for industry and regulator representatives, this next round began to sort out some detail. On November 20th and December 11th, 2020, public stakeholder meetings were held on the Prescription Drug User Fee Amendments reauthorization (PDFUA VII) that will span the fiscal years 2023-2027. Broadly, the November 20th meeting presented information on the FDA’s drug safety system, while the December 11th meeting focused largely on the increasing resource needs of cell and gene therapy.
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With just days remaining before the transition to a new administration, the Department of Health and Human Services (HHS) is pushing through policies that could impact the US FDA regulatory oversight. Last week, HHS announced it had finalized a rule that would cause its regulations to expire if they are not reviewed every 10 years, with some exceptions provided for medical device-specific regulations, food standards and over-the-counter drugs, among other carveouts. HHS also announced that it was issuing two new policies aimed at lifting premarket review requirements for some medical devices and imposing reporting requirements on FDA pertaining to its review timelines for drugs.
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Three strategic initiatives form the backbone of the regulatory science areas of focus for the US FDA in 2021, according to a new report. The Focus Areas of Regulatory Science (FARS) issued this week “outlines topics that the FDA has identified as needing continued targeted investment in regulatory science research to facilitate the development of innovative products, provide data and methodology to inform regulatory decision-making and improve guidance to sponsors,” according to FDA Chief Scientist Denise Hinton. The first of the three broad themes, is dubbed “Unleashing the Power of Data.” The second theme focuses on using innovation to increase choice and competition, while the third is aimed at empowering patients and consumers.
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The US FDA’s Center for Drug Evaluation and Research has detailed plans to reorganize five of its offices this year. In an email to staff, Acting CDER Director Patricia Cavazzoni said the approved structural changes in the Office of Generic Drugs (OGD), Office of Strategic Programs (OSP), Office of Surveillance and Epidemiology (OSE), Office of Management and Office of Compliance “will enable us to respond to new priorities more effectively and efficiently.” The announcement follows a multi-year effort by CDER to modernize its new drug program that led to the reorganization of the Office of New Drugs that wrapped up last year.
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The US FDA has issued draft guidance on the development, testing, and trial design for human gene therapies for neurodegenerative diseases. The document, released this week, also highlights approval pathways for these novel products. The draft guidance, which applies to products for both adult and pediatric populations, emphasizes the importance of early communication with the FDA before the submission of an investigational new drug (IND) application. The agency pointed to INTERACT meetings, which can be used to discuss issues in a product’s early preclinical program, and pre-IND meetings, which occur later in development but prior to the submission of an application.
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EMA has recommended granting a conditional marketing authorization for COVID-19 Vaccine Moderna to prevent Coronavirus disease (COVID-19) in people from 18 years of age. This is the second COVID-19 vaccine that EMA has recommended for authorization. EMA’s human medicines committee (CHMP) has thoroughly assessed the data on the quality, safety and efficacy of the vaccine and recommended by consensus a formal conditional marketing authorization be granted by the European Commission. This will assure EU citizens that the vaccine meets EU standards and puts in place the safeguards, controls and obligations to underpin EU-wide vaccination campaigns.
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Recognizing the pace at which drug developers are moving ever further into individualized medicine, the US FDA has issued a draft guidance addressing submission processes for some hyper-specialized treatments. The new document, which gives high-level guidance for investigational new drug (IND) submissions of individualized anti-sense oligonucleotides (ASOs), takes into consideration that these “N of 1” therapies come with “a set of challenges and considerations not seen with the typical drug intervention,” according to a statement from Patrizia Cavazzoni, MD, the acting director of FDA’s Center for Drug Evaluation and Research.
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With a final push that saw the regulator approving its final drug on December 23rd, 2020 the U.S. FDA’s CDER finished the year with 53 approvals for new molecular entities and new therapeutic biological products. Making 2020 one of the agency’s best years for annual approvals. That being said, it should be noted that this count does not include vaccines, allergenic products, blood and blood products, plasma derivatives, cellular and gene therapy products, or other products approved in 2020 by the Center for Biologics Evaluation and Research.
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The MHRA has published guidance on the Innovative Licensing and Access Pathway (ILAP) it is setting up to accelerate time to market. Drug developers that follow the pathway will benefit from more input from MHRA and stakeholders including the National Institute for Health and Care Excellence. Drug developers can access the pathway as soon as they have nonclinical data on a new chemical entity, biological medicine, new indication or repurposed molecule, although the scheme also permits entry once a candidate is somewhat closer to market. MHRA is encouraging companies to apply early in development to maximize the benefits.
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A “large number” of centrally authorized products (CAPs) contain UK sites and activities that need to be removed by early next year due to Brexit. Companies have until the end of February to submit the required Type IA variation. The European Medicines Agency (EMA) identified the continued presence of UK sites and activities on the marketing authorization dossiers of CAPs as an area in which the industry still needs to act on a recent webinar. EMA used the webinar to discuss preparations for the end of the transition period that has kept the UK subject to EMA rules in recent months.
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The US FDA this week finalized guidance for sponsors on interacting with the agency on complex innovative trial designs (CID) for drugs and biologics. The nine-page final guidance comes just over a year after the draft version was released for comment and two years after the agency launched its CID pilot program. The aim of the CID program is to facilitate the use of novel and innovative trials, including complex adaptive and Bayesian designs that often feature simulation. The FDA explains that “there is no fixed definition of CID because what is considered innovative or novel can change over time.”
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The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended 15 medicines for authorization following its December meeting. Among the medicines are eight new drugs and biologics, four generics, two biosimilars and one hybrid medicine. The new drugs included three products with orphan designation: AstraZeneca’s Lumoxiti, Celgene’s Inrebic and Advicenne’s Sibnayal. Following an accelerated assessment, the committee recommended conditional marketing authorization for Daiichi Sankyo’s Enhertu and Eli Lilly’s Retsevmo.
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The COVID-19 vaccine candidate developed by Sanofi and GSK hit a snag that will delay the launch of a Phase III study due to an insufficient immune response in older trial patients. The vaccine will now not be available until the end of 2021 – if at all. According to Sanofi the interim results of its Phase I/II vaccine study showed an immune response comparable to patients who recovered from COVID-19 in adults aged 18 to 49 years. However, in adults 50 and above, there was an “insufficient immune response.” Instead of a Phase III trial, Sanofi said it will plan a Phase IIb study with an improved antigen formulation that refines the concentration of antigen in order to provide high-level immune response across all age groups.
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The US FDA has authorized Pfizer and BioNTech’s COVID-19 vaccine for emergency use in individuals ages 16 and older just one day after the agency’s advisory panel voted 17-4 in favor of the vaccine. Now that the vaccine has been authorized just three weeks after Pfizer and BioNTech submitted their emergency use authorization (EUA) request, a nationwide rollout of the vaccine is set to begin. In the coming days, 2.9 million of the US’ initial supply of 6.4 million doses of the mRNA vaccine will be distributed to the states. Another 2.9 million doses will be withheld for the booster dose of the two-injection regimen and an additional 500,000 are being reserved for unforeseen circumstances.
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The benefits of the COVID-19 vaccine developed by Pfizer and the German firm BioNTech outweigh potential risks for people aged 16 years and up, according to a US FDA advisory committee. The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted 17-4, with one abstention, to endorse the favorable benefit-risk profile put forward as the lone voting question for the committee’s meeting. This vote paves the way for imminent issuance of an emergency use authorization (EUA) for the vaccine; the vaccines advisory committee of the Centers for Disease Control and Prevention (CDC) is holding a split meeting this afternoon and Sunday.
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The US FDA has issued a pair of draft guidance documents aimed at guiding the selection of proprietary names for prescription and non-prescription drugs. The best practices set forth in the guidances are designed “to help minimize proprietary name-related medication errors and otherwise avoid adoption of proprietary names that contribute to violations of the Federal Food, Drug, and Cosmetic Act (FD&C Act) and its implementing regulations,” according to the guidances. The best practices documents apply only to human drugs; the non-prescription drug naming guidance runs to 17 pages, whereas the guidance dedicated to prescription drug naming is 42 pages long.
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The FDA concluded in a detailed analysis that the first Covid-19 vaccine being considered for U.S. distribution “met the prescribed success criteria” in a clinical study, paving the way for the agency to green-light distribution as early as this weekend. An outside panel of scientific advisers will review the FDA report this Thursday, along with a companion analysis from the vaccine’s manufacturers, Pfizer and German partner BioNTech. A favorable recommendation from the panel is expected to be followed within a few days by the FDA granting emergency authorization for the vaccine.
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The US FDA has kept its new drug approvals for 2020 on track to match or best approvals in 2019, despite the COVID-19 pandemic. By December 4th, 2020, the FDA had amassed a list of 48 new molecular entities and new therapeutic biological products approved by the agency’s Center for Drug Evaluation and Research (CDER), equaling the total for 2019. Of those 48 new entities approved by the agency to date in 2020, 15 were for cancer therapies. As of this writing a number of applications still sit before the FDA with PDUFA action dates between now and the end of this year.
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It isn’t easy to be a highly effective regulatory writer, but clear, well-written documentation is a critical part of bringing essential medicines and medical products to market. In RAPS’ newly released, Regulatory Writing: An Overview, Second Edition, aspiring and experienced writers alike will find expert guidance and advice that is easy to apply. Completely updated from the previous edition, Regulatory Writing covers content development for regulatory documents in various specialties, including chemistry, manufacturing, and controls (CMC), clinical, nonclinical, labeling, vaccines, lay summaries, and publications. The book includes 30 chapters from regulatory writing experts with a diverse base of knowledge and extensive direct experience.
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Developers of combination products have new final guidance from the US FDA about requesting feedback from the agency during the full product development cycle. In response to feedback received during the consultation period on the January 2020 draft guidance, the FDA has adjusted the final guidance to give developers more information about the best use of combination product agreement meetings (CPAMs) as well as how agreements reached through CPAMs are structured and what they contain. The guidance is issued as part of 21st Century Cures Act mandates that require the FDA to provide sponsors of combination products with final guidance.
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The UK’s MHRA granted temporary authorization this week to Pfizer and BioNTech’s mRNA-based BNT162b2 COVID-19 vaccine, vaulting ahead of regulators in the US and EU. The temporary authorization is “batch-specific” and applies only to a single batch of the vaccine. Subsequent batches must be approved by MHRA and must meet the same authorization conditions as the initial batch. MHRA said the decision was made based on advice from its Commission on Human Medicines (CHM) following a rolling review of data submitted by the companies. The agency is currently conducting rolling reviews of vaccine candidates from Moderna and AstraZeneca. The UK’s quick decision set off a dispute with its EU counterparts over whose review approach is more appropriate and amplified pressure on the US FDA to issue an emergency use authorization (EUA) for the vaccine.
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An international coalition of regulators has issued a statement in support of continuing clinical trials for vaccines against COVID-19 “for as long as is feasible,” but ideally for at least one year after vaccination. The International Council of Medicines Regulatory Authorities (ICMRA) issued the statement in part to address a thorny ethical dilemma to be faced by vaccine sponsors and healthcare professionals when a vaccine is authorized: Should trial participants be unblinded as to their allocation, and placebo recipients offered a vaccine? Recognizing the importance of high-quality clinical data in regulatory and clinical decision-making about COVID-19 vaccines, the ICMRA statement notes that regulators are ready to make speedy decisions with the results that come before them for approval decisions.
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The US FDA this week announced it will pilot a new program it hopes will spur the development of novel drug development tools (DDTs) that do not fit within the agency’s existing DDT qualification programs. The qualification process for DDTs was revamped under the 21st Century Cures Act and FDA currently offers specific qualification programs for biomarkers, clinical outcome assessments (COAs) and animal models for use under the Animal Rule, which allows for the approval of drugs based on data from animal studies when human efficacy studies are not ethical or feasible. Under the agency’s new Innovative Science and Technology Approaches for New Drugs (ISTAND) program, the FDA says it will offer developers early feedback on proposed DDTs that are “out of scope for existing DDT qualification programs” and provide a path to qualification or other outcomes when qualification is not appropriate.
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The FDA has authorized one of the first Covid-19 tests that measures the amount of neutralizing antibodies produced by the body’s immune system after exposure to the virus — a “new generation” of coronavirus test that is designed to tell how well someone is protected against infection. Unlike many previously authorized antibody tests that can tell if you’ve had the virus or not, the COVID-SeroKlir test measures your specific levels of Covid-19 neutralizing antibodies. The FDA has previously authorized some tests that estimate the level of antibodies, but not specifically for neutralizing antibodies. The agency cautioned that much remains unknown about the nature of Covid-19 neutralizing antibodies in humans and that a high level of antibodies does not necessarily guarantee immunity against the virus.
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AstraZeneca says that it will submit preliminary data from large clinical trials in the U.K. and Brazil to the FDA as part of an application for emergency authorization. Data from those trials show the vaccine was about 70 percent effective across two different dosing regimens. When the vaccine was given as a half dose and then a full dose one month later to about 2,700 participants, it had an efficacy of 90 percent. When given as two full doses at least one month apart to about 8,900 participants, it showed 62 percent efficacy. The company says it will work with the FDA to adjust the design of its late-stage U.S. trial to test the half-dose regimen, rather than the higher dose that proved less effective in the U.S and Brazil studies.
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In a recently issued draft questions and answers guidance the US FDA offers insights into how it will handle certain aspects of submissions and labeling for interchangeable biosimilars. When finalized, the FDA will move the questions and answers from the draft guidance to its companion final questions and answers guidance on biosimilar development. In the first of four questions and answers in the draft guidance, the FDA explains how it anticipates handling applications for interchangeable biosimilars that include data to support licensure as a biosimilar but not as an interchangeable product.
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Washington, D.C., USA- January13, 2020: FDA Sign at its headquarters in Washington. The Food and Drug Administration (FDA or USFDA) is a federal agency of the USA.

At the kickoff meeting for the reauthorization process for the Biosimilar User Fee Act (BsUFA III) program, the US FDA and industry shared their wish lists for the third iteration of the agency’s biosimilars review program. While the FDA laid out four broad goals for the FY2023-2027 program, industry presented more specific proposals, including the creation of a new pre-development meeting and issuance of further guidance on interchangeable biosimilars. The meeting, which sets the stage for the months long negotiations between the FDA and regulated industry, began with an overview of the BsUFA program by Patrizia Cavazzoni, acting director of CDER and Andy Kish, director of the Office of Program and Strategic Analysis at CDER.
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The EMA has refreshed its points to consider document on remote pharmacovigilance inspections during a crisis, such as the current COVID-19 pandemic. The revisions to the document are minor and mostly relate to the preparation phase ahead of a pharmacovigilance inspection when an on-site inspection would not be feasible due to a crisis. In the revised document, EMA has replaced the term “distant/virtual” with “remote” and has expanded its explanation of a crisis situation that would prevent an on-site inspection from being conducted. One of the biggest changes to the document is the emphasis on the need to determine whether a marketing authorization holder has the technical capabilities to facilitate remote access to their electronic systems and maintain communication with inspectors.
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As the time draws near for the leaders in the global race for a vaccine against COVID-19 to submit emergency use authorization (EUA) authorizations to the US FDA, the agency has proclaimed a new commitment to transparency. With a stated goal of disclosing information from COVID-19-related EUA review documents “as appropriate and consistent with our longstanding practice of posting scientific reviews after new drug and biological product approvals,” the FDA also reserved to itself the ability to redact trade secrets or other information that would be exempt from disclosure under the Freedom of Information Act (FOIA) or protected from disclosure by other legislation.
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The U.S. FDA stunned Sanofi with a Complete Response Letter (CRL) for its blood cancer treatment sutimlimab. The medication was expected to be the first treatment approved for the treatment of cold agglutinin disease (CAD). The regulatory agency is understood to have rejected the medication due to deficiencies from a pre-license inspection of a third-party manufacturing facility. In its CRL, the FDA said there were no clinical or safety deficiencies with respect to the application. Sanofi said the BLA cannot be approved until there are “satisfactory resolutions of the observations by the third-party manufacturer.”
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In its November meeting, a committee of the European Medicines Agency gave its second thumbs down to the monoclonal antibody Gamifant (emapalumab), confirming a July 2020 negative opinion on the Novimmune SA drug. The US FDA approved Gamifant for pediatric use in primary hemophagocytic lymphohistiocytosis in November 2018; this was the same indication for which EMA has twice declined authorization. The EMA’s human medicines committee (CHMP) had positive recommendations on the first go for four new medicines: Roclanda (latanoprost/netarsudil), Xofluza (baloxavir marboxil), Phesgo (pertuzumab/ trastuzumab), and Onbevzi (bevacizumab).
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The Senate Committee on Appropriations this week released draft legislation proposing $3.21 billion in discretionary funding for the US FDA for FY2021, closely matching the amount the agency would receive under the minibus passed in the U.S. House of Representatives in July. In total, the draft bill would set FDA’s total FY2021 funding, including user fees, at $5.88 billion. As with the House bill, the draft Senate legislation would provide the FDA with $40 million in additional funding over the FY2020 enacted level and would release an additional $70 million in 21st Century Cures Act funds. Learn More

U.S. Senators Elizabeth Warren and Tina Smith are concerned about the safety of pharmaceuticals and drug components coming into the United States, as the coronavirus pandemic has put a halt to many inspections of overseas drug manufacturing facilities. In a letter to FDA Commissioner Stephen Hahn released this week, the senators wrote: “We are concerned that, absent proper oversight of our drug supply chain and the overseas facilities manufacturing products millions of Americans rely on, patients will face an increased risk of drug contamination or other problems with quality, purity, or potency.” Right now, inspections are only happening on a case-by-case basis and are limited to those described as mission-critical, pre-approved, or for cause. Learn More

The US FDA has granted an emergency use authorization for an outpatient therapy to treat mild to moderate COVID-19. The monoclonal antibody, bamlanivimab, is authorized for outpatient use in adults and pediatric patients. The treatment is reserved for patients who have positive direct SARS-CoV-2 viral tests, not antigen tests, and who are “at high risk for progressing to severe COVID-19 and/or hospitalization,” according to the FDA’s November 9th letter of authorization issued to bamlanivimab’s sponsor, Eli Lilly and Company. The conditions of the EUA granted to Lilly include a specific directive to retain an independent third party to review batch records and “any underlying data and associated discrepancies” of the drug substance. Learn More

Novavax has announced that the U.S. FDA has granted Fast Track Designation for NVX-CoV2373, the Company’s COVID-19 vaccine candidate. Currently in late-phase clinical development, NVXCoV2373 is a stable, prefusion protein made using Novavax’ nanoparticle technology and includes its proprietary MatrixM™ adjuvant. Novavax expects to begin its pivotal Phase 3 clinical trial in the United States and Mexico by the end of November. Data from the event-driven trial could support global authorization and approval, including in the U.S. The Company’s ongoing Phase 3 clinical trial in the UK to evaluate the efficacy, safety and immunogenicity of NVX-CoV2373 is expected to be fully enrolled by the end of November. Learn More

An advisory committee to the US FDA handed the agency a resounding thumbs down not only on the Alzheimer’s disease candidate therapy aducanumab, but on the agency’s framing of the questions put before the committee and the contents of its briefing documents. The advisory committee heard from the FDA and from the sponsor, Biogen, in separate presentations, but an integrated briefing document was presented that, in the minds of some committee members, made it difficult to parse out the FDA’s perspective and that of the sponsor. The FDA is not obligated to follow the recommendations of its advisory committees, though it usually does. Learn More

Expect new guidance from the US FDA on the manufacture of CAR-T cells, said Peter Marks, director of the agency’s Center for Biologics Evaluation and Research (CBER). Marks answered questions on a range of issues related to cell and gene therapy development during a webinar hosted by Genetic Engineering & Biotechnology News. Saying that CBER is working on new guidelines for CAR T-cell therapy manufacturing, Marks said, “The agency is trying to put out some more clear information on what we’re expecting for CAR T-cells. I’m not sure exactly when it’s going to appear, but stay tuned,” Marks said. Learn More

While the world has largely been focused on the development of vaccines and therapeutics for COVID-19, the U.S. FDA has remained busy lining up potential approvals of medications for other diseases and illnesses. Many of these drugs have been granted Fast Track designation over the past several weeks, which is intended to expedite the review of these treatments. BioSpace takes a look at some of the most recent medications to receive this designation. Learn More

As the buzz builds around messenger RNA (mRNA) technology’s use for two leading COVID-19 candidates, manufacturers and regulatory professionals are facing facts: This is not simple technology. Complex manufacturing processes, delivery vehicles that must be treated more as drug substances than excipients, and potential immunogenicity headaches are among the challenges industry faces as this promising technology is harnessed to address an increasing number of health conditions. Messenger RNA is considered by both the US FDA and the EMA to be gene therapy even though RNA does not interact with the genome. However, mRNA, which is regulated by the FDA’s CBER is not yet classified as a regenerative medicine advanced therapy (RMAT). EMA considers mRNA to be an advanced therapy medicinal product (ATMP). Learn More

To comply with a recent executive order, the US FDA has released a list of more than 300 essential drugs and medical devices that will serve as a basis for prioritized federal procurement of those products from domestic sources. The list was developed in response to President Donald Trump’s “Buy American” executive order, issued in August, aimed at boosting domestic production of drugs and medical devices. In total, the list includes 223 drugs and biological products and 96 medical devices and in vitro diagnostics deemed “medically necessary to have available at all times,” in adequate supply and in appropriate dosage forms. The list also specifies the critical inputs, such as API’s or sensor components, necessary to produce each product. Learn More

Reflecting the extraordinary circumstances of the pandemic, the EMA has announced that the agency is speeding announcements regarding the progress of clinical trials of vaccines and therapeutics for COVID-19. The agency is also making public certain information about the progress of clinical trials that would not ordinarily be published. Perhaps the most notable departure from business as usual is publication of full deidentified clinical trial data for COVID therapeutics and vaccines after they receive marketing authorization, a move EMA had announced in September. Learn More

Researchers at the US FDA found that, for drugs that are new chemical entities, generic submissions were more likely when the reference brand-name drug generated more revenue. Looking into other factors that predict that an abbreviated new drug application (ANDA) will be filed for a reference drug, the investigators also found that sponsors were less likely to file ANDAs for complex drugs. In a retrospective study examining 400 brand-name drugs eligible for ANDA filing between 2011 and 2017, investigators at FDA's CDER identified 140 that had originally been approved as new chemical entities (NCEs). Learn More

Trust means everything. Trust in vaccines facilitated the incredible positive impact that vaccination had on public health in reducing illness and death over the past century. In the middle of a global pandemic, it is precisely a safe and effective COVID-19 vaccine that will help bring life back to normal if people are willing to receive the vaccine because they have confidence in it. Therefore, it is critical to be open and transparent about the process that the U.S. Food and Drug Administration will follow to help make safe and effective COVID-19 vaccines available. Learn More

Generic drugmakers have received finalized guidance from the US FDA. In finalizing a 2017 draft, the agency gives sponsors of abbreviated new drug applications (ANDAs) information about how to identify reference listed drugs (RLDs), further details on reference standards, and what forms the basis for an ANDA submission. In clarifying how to identify the basis of submission and reference standard, as well as how to identify RLDs, the FDA acknowledged in the Federal Register announcement of the guidance's availability that "A variety of factors has led to confusion among stakeholders on what these terms mean and how an ANDA applicant should use them." Learn More

The US FDA has deferred a decision on Spectrum Pharmaceuticals' SPPI.O drug candidate to treat chemotherapy induced loss of white blood cells in cancer patients due to COVID-19 related travel curbs. This comes after the regulatory agency was unable to inspect the company's South Korea-based manufacturing plant, a step required before the approval of the drug, the company said. The regulator had warned in April it may not be able to sustain its current level of timely reviews and approvals of marketing applications as many of its staff were allocated to the coronavirus crisis. Learn More

Clinical trials for the AstraZeneca Oxford coronavirus vaccine, AZD1222, have resumed across the world with regulators in the US, UK, Brazil, South Africa and Japan confirming that it was safe to do so. The FDA authorized the restart in the US, following the resumption of trials in other countries in recent weeks. The FDA reviewed all safety data from trials globally and concluded it was safe to resume the trial. As part of the standard review process for trial safety events, a voluntary pause to vaccination across all global trials was triggered on September 6th to allow the examination of safety data by independent monitoring committees. Learn More

After a vaccine against COVID-19 is authorized or approved, how will its safety and efficacy be assessed? In addition to ongoing postmarket surveillance requirements, the US FDA and the CDC will strive for near real-time safety and efficacy surveillance of COVID-19 vaccines. The FDA is currently developing master protocols to guide its safety and effectiveness oversight, said an FDA official during the FDA's first COVID-19 vaccine-focused advisory committee hearing. "We'll be posting the protocols for public comment," said Stephen Anderson, PhD, director at the FDA's Center for Biologics Evaluation and Research (CBER). Learn More

After four straight years of posting record numbers of abbreviated new drug application (ANDA) approvals, the US FDA has broken its streak, approving 22% fewer generic drugs in FY2020 than it did in FY2019. In FY2020, the FDA approved a total of 909 ANDAs, 737 full approvals and 172 tentative approvals, compared to a record 1,171 ANDAs in FY2019. Complete responses also declined to 2,010 compared to 2,310 in FY2019. The dip in approvals comes after the FDA increased its focus on generic drug approvals as part of the drug competition action plan championed by former FDA Commissioner Scott Gottlieb and three years into the second Generic Drug User Fee Amendments (GDUFA II) program. Learn More

The U.S. health regulator's criteria for allowing emergency use of a COVID-19 vaccine and plans to monitor its safety after a regulatory go-ahead, are among the topics to be discussed at a closely watched meeting scheduled for Thursday. Details posted on the FDA website showed the U.S. Centers of Disease Control and Prevention has formulated plans to monitor safety and effectiveness of a vaccine even after the FDA allows for its emergency use. The FDA panel would make recommendations at the end of the meeting, according to the agenda, but did not specify details. Learn More

Two new pilot programs related to manufacturing quality management have been launched by the US FDA. The agency's Center for Drug Evaluation and Research (CDER) is hoping to "gain insight from third-party assessments of a manufacturer's quality management system to inform future development of an FDA rating system," according to CDER's announcement to industry of the new pilot programs. The rating system that FDA hopes to develop from the pilot programs would better allow an apples-to-apples comparison of facilities, which could be of benefit to a variety of stakeholders including healthcare facilities and consumers. The initiative is part of the broader FDA effort to shift to risk-based pharmaceutical quality assessment systems. Learn More

With negotiations underway for the next iterations of the US FDAs new and generic drug user fee programs, top officials from the Center for Drug Evaluation and Research (CDER) discussed budget and staffing priorities in a call with the Alliance for a Stronger FDA. "We have really made major strides thanks to Congress' support in the appropriations that we received in 2019, in investing in modernizing the platforms that support the review of applications and the data analytics platforms that we intend to build and refine to allow us to communicate with the external world, including sponsors and researchers, using cloud-based technologies," said CDER Acting Director Patrizia Cavazzoni. Learn More

While the US FDA is still receiving investigational new drug applications (INDs) for cell and gene therapies, officials are concerned about the impact of the COVID-19 pandemic on clinical trials. "It's clear that COVID-19 has adversely affected all aspects of development of cell and gene therapies," said Peter Marks, director of FDA's Center for Biologics Evaluation and Research (CBER), while speaking at the Alliance for Regenerative Medicine's Meeting on the Mesa. "For some of the studies that are ongoing there are some real challenges to overcome in terms of endpoints that may have been missed." The pandemic also has disrupted global harmonization efforts around gene therapies, Marks said. Learn More

Biosimilars have been slow to enter the US market for complex reasons ranging from the manipulation of patent protections to brand-name drug rebates. Now a new analysis, published in the Mayo Clinic Proceedings, suggests that a "high testing bar" for biosimilars to gain regulatory approval is another contributing factor. The analysis found that the median time from the start of a Phase I study to approval by the US FDA was more than 5 years. As of May 1st, 2020, the FDA has approved just 26 biosimilars, of which 17 products have been marketed. In contrast, the EMA has approved 64 biosimilars. Learn More

U.S. drug inspectors uncovered serious quality control problems at an Eli Lilly and Co pharmaceutical plant that is ramping up to manufacture one of two promising COVID-19 drugs according to government documents and three sources familiar with the matter. The three sources who spoke to Reuters requested that their names be withheld so they could speak freely without fear of retaliation. Inspectors who visited the Lilly plant in Branchburg, New Jersey, last November found that data on the plant's various manufacturing processes had been deleted and not appropriately audited, government inspection documents show. Following its November inspection, the FDA issued an "Official Action Indicated" (OAI) notice. Learn More

Avenue Therapeutics has announced that it has received a Complete Response Letter ("CRL") from the U.S. FDA regarding the Company's New Drug Application ("NDA") for IV tramadol. The CRL stated that although the pivotal Phase 3 clinical trials demonstrated statistically significant outcomes for all of the primary and many secondary endpoints, the FDA has determined that it cannot approve the application in its present form. The CRL stated that IV tramadol, intended to treat patients in acute pain who require an opioid, is not safe for the intended patient population. The FDA noted specific concerns of potential for opioid "stacking" and associated increased likelihood of adverse effects. Learn More

The US Government Accounting Office (GAO) found that many elements of an effort to improve laboratory safety at the U.S. FDA have not been met, over three years after establishing an Office of Laboratory Safety. For example, the safety oversight office still cannot perform unannounced laboratory inspections, according to the report. Prompted by the 2014 discovery of boxes of smallpox virus that were improperly stored in a laboratory cold room maintained by FDA on the campus of the National Institutes of Health, GAO began an investigation that led to a series of five recommendations made in 2016 aimed at improving the agency's laboratory safety. Learn More

Two days after the US FDA's surprise publication of guidance setting criteria for emergency use authorization (EUA) for COVID-19 vaccines, Peter Marks, director of the Center for Biologics Evaluation and Research shared some of the thought process that went in to its recommendations. One of the most significant recommendations in the guidance is FDA's insistence on a median of two months of follow-up of Phase 3 trial participants following their last dose. Speaking at the Food and Drug Law Institute's annual conference, Marks explained that FDA settled on a median of two months because that would provide data on enough patients for enough time to be able to observe most of the events that concern the agency. Learn More

The European Medicines Agency (EMA) is transitioning to an online platform for drug developers to use when requesting scientific advice. The change will become effective on October 19th for developers of both human and veterinary medicinal products. The regulatory and scientific information management platform, known as IRIS, "aims to make the handling of product-related regulatory procedures more efficient and user-friendly and to ensure better data quality through integration with other EMA systems," including EMA's substance, product, organization and referential (SPOR) portal, said EMA. Learn More

Amid reports that the White House had blocked stricter guidance from the US FDA on emergency use authorizations (EUAs) for COVID-19 vaccines, the agency has issued guidance hours after disclosing some of its advice to vaccine makers in briefing documents for an upcoming advisory committee meeting. The FDA appears to have sidestepped the White House by releasing five pages of advice given to vaccine makers on COVID-19 vaccine EUAs in briefing documents for its upcoming Vaccines and Related Biologics Advisory Committee meeting. Hours later, in a surprise move, the FDA released its COVID-19 vaccine EUA guidance in full. Learn More

The US FDA has announced historic low rates for the fiscal year 2021 fees to use its three priority review voucher programs. For the upcoming fiscal year, the rate to use a material threat medical countermeasure (MCM) priority review voucher (PRV) is an all-time low $1,360,879. The PRV rate is the same for the rare pediatric disease and rare tropical disease voucher programs, according to Federal Register announcements. This figure is in addition to the required prescription drug user fee amendments fees for FY 2021, and it represents the agency's estimate of the additional cost for the compressed 6-month review timeline earned by presentation of the PRV. Learn More

Mesoblast has made public a decision by the U.S. to issue a Complete Response Letter to its Biologics License Application (BLA) for remestemcel-L for the treatment of pediatric steroid-refractory acute graft versus host disease (SR-aGVHD). While the Oncologic Drugs Advisory Committee (ODAC)1 of the FDA voted 9:1 that the available data support the efficacy of remestemcel-L in pediatric patients with SR-aGVHD, the FDA recommended that Mesoblast conduct at least one additional randomized, controlled study in adults and/or children to provide further evidence of the effectiveness of remestemcel-L for SR-aGVHD. Learn More

Solid Biosciences has announced that the U.S. FDA has lifted the clinical hold placed on the Company's IGNITE DMD Phase I/II clinical trial. The news helped drive the company's share price up roughly 70% in trading. As announced in July 2020, the FDA had requested further manufacturing information, updated safety and efficacy data for all patients dosed, and provided direction on total viral load to be administered per patient. Based on the Company's response to these requests, the FDA acknowledged that the Company satisfactorily addressed all clinical hold questions. Learn More

The U.S. FDA has broadened its investigation of a serious illness in AstraZeneca's COVID-19 vaccine study and will look at data from earlier trials of similar vaccines developed by the same scientists, three sources familiar with the details told Reuters. AstraZeneca's large, late-stage U.S. trial has remained on hold since Sept. 6, after a study participant in Britain fell ill with what was believed to be a rare spinal inflammatory disorder called transverse myelitis. The widened scope of the FDA probe raises the likelihood of additional delays for what has been one of the most advanced COVID-19 vaccine candidates in development. The requested data was expected to arrive this week. Learn More

Speaking at the Financial Times' US Pharma and Biotech Summit this week, US FDA Commissioner Stephen Hahn said the agency will stick to the standards and criteria it has laid out to approve or authorize applications for vaccines to prevent coronavirus disease (COVID-19). In recent weeks, Hahn has made numerous statements asserting that the agency will not approve or authorize a vaccine that does not meet the agency's "rigorous expectations," despite repeated claims by President Donald Trump that a vaccine could be greenlighted before the November election. Learn More

The U.S. FDA has put a hold on Inovio Pharmaceuticals plans to start final trials of its coronavirus vaccine as the agency seeks more information, including details on a delivery device used to inject genetic material into cells. The mid-to-late trials, which were awaiting approval from the U.S. FDA, were scheduled to start this month after they were postponed from this summer. The drug developer said this week that the latest delay due to the FDA's "partial clinical hold" was not due to any side effects in the early-stage study of the vaccine, which was continuing. Learn More

The US Department of Health and Human Services (HHS) has issued a final rule that clears a path for importation of some Canadian drugs into the US. The measure follows a July 24th, 2020 executive order issued by President Donald Trump that directed the HHS secretary to take action to expand access to lower cost imported drugs. In a September 24th press release, the US FDA said that the final rule would help affordability of drugs for US patients. The final rule allows states, Indian tribes and potentially pharmacies and wholesalers to seek FDA authorization of importation programs. Learn More

President Donald Trump claimed this week that the White House could override the US FDA if the agency released tougher standards for the authorization of a Covid-19 vaccine, casting such a move as "political." His comments come as the FDA considers new Covid-19 vaccine guidelines that would likely push an authorization beyond Election Day, according to three sources familiar with the situation. That timeline would dash Trump's hopes of a pre-election authorization, having repeatedly said the vaccine could be ready by November 3. "We're looking at that and that has to be approved by the White House. We may or may not approve it," the President said of the new FDA guidelines. Learn More

Testifying before the Senate Health, Education, Labor and Pensions Committee this week, US FDA Commissioner Stephen Hahn said that politics will not play a part in the decision to approve or authorize a vaccine for coronavirus disease (COVID-19). Hahn's reassurances come amid reports from the Washington Post, Financial Times and New York Times that the agency is preparing to issue guidance on its expectations for a COVID-19 vaccine EUA that will ask for a median two months of monitoring after Phase 3 trial participants receive their last dose, making an EUA for a vaccine before the election unlikely. Hahn and CBER Director Peter Marks have both previously said that such guidance is coming. Learn More

Investigators of new drugs must continue to send safety reports of serious and unexpected adverse events to their institutional review boards during the public health emergency of the novel coronavirus pandemic, according to an updated guidance from the US FDA. The updated information came on September 21st, 2020 in the form of a new addition to the questions and answers in the FDA's guidance on conduct of clinical trials of medical products during the COVID-19 public health emergency. The guidance, originally issued in March 2020, has been expanded and updated throughout the pandemic. Learn More

As trust in federal health agencies has withered over the last few months, a group of Black physicians has been working on an antidote: creating their own expert task force to independently vet regulators' decisions about Covid-19 drugs and vaccines as well as government recommendations for curbing the pandemic. Organized by the National Medical Association - founded in 1895 as an answer to racist professional societies excluding Black doctors - the committee is meant to safeguard against any unscientific guidance from the Centers for Disease Control and Prevention and the Food and Drug Administration. Learn More

In a stunning declaration of authority, Alex M. Azar II, the secretary of health and human services, this week barred the nation's health agencies, including the Food and Drug Administration, from signing any new rules regarding the nation's foods, medicines, medical devices and other products, including vaccines. Going forward, Mr. Azar wrote in a Sept. 15 memorandum obtained by The New York Times, such power "is reserved to the Secretary." The bulletin was sent to heads of operating and staff divisions within H.H.S. It's unclear if or how the memo would change the vetting and approval process for coronavirus vaccines, three of which are in advanced clinical trials in the United States. Learn More

The European Medicines Agency is on the verge of releasing revised guidance for advanced therapy medicinal products containing genetically modified cells, which includes chimeric antigen receptor (CAR)-T cell therapies. The "Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells" was originally issued in 2012 but underwent revision and consultation from July 2018-July 2019. The revised version is expected to be adopted in October and published in November, according to Ana Hidalgo-Simon, MD, PhD, head of advanced therapies at EMA. Learn More

Investigators should use a patient-reported outcomes approach, with an emphasis on electronic data collection, in their research on therapies to treat or prevent COVID-19, according to a new guidance from the US FDA. The guidance, which is effective immediately during the public health emergency of the coronavirus pandemic, outlines how to capture disease-related symptoms in clinical trials focusing on COVID-19 prevention or treatment for outpatient adults and adolescents. Learn More

Health and Human Services Secretary Alex Azar led an escalating pressure campaign against his own Food and Drug Administration this spring and summer, urging the agency to abandon its responsibility for ensuring the safety and accuracy of a range of coronavirus tests as the pandemic raged. Then in late August, Azar took matters into his own hands. Overriding objections from FDA chief Stephen Hahn, Azar revoked the agency's ability to check the quality of tests developed by individual labs for their own use, according to seven current and former administration officials with knowledge of the decision. Learn More

A Phase 1 trial assessing Cellectis' investigational CAR T-cell therapy, UCARTCS1A, has been placed on hold by the U.S. FDA. The MELANI-01 study (NCT04142619) was assessing the safety and clinical activity of different doses of UCARTCS1A, administered in a single infusion, with the aim of determining the optimal dose for further testing. The clinical hold came after one patient, who received the second dose level of the therapy, died from cardiac arrest during the study. Cellectis is working closely with the FDA to lift this clinical hold, which will require the company to make changes to the study's protocol to enhance participants' safety. Learn More

A new temporary guidance for drug and biologics manufacturers from the US FDA details recommendations for returning to normal production operations during the public health emergency of the COVID-19 pandemic. "This guidance provides recommendations to help manufacturers prioritize products as they resume normal operations and as they remediate current good manufacturing practice (CGMP) activities that were necessarily delayed, reduced, or otherwise modified during the public health emergency in order to maintain production and the drug supply," said FDA in announcing the availability of the temporary guidance. Learn More

The head of the US FDA, Commissioner Stephen Hahn, said he has "no intention" of overruling career scientists at the agency on an approval of a coronavirus vaccine even though he has the authority. He went on to add that any vaccine data released will be discussed publicly by an advisory committee of outside experts and that Peter Marks, who runs the FDA division that oversees vaccine approvals, will ultimately make a recommendation. "I have complete and absolute confidence in the scientists at the FDA and the decision-making that they have here," he added. Learn More

Manufacturers can request emergency use authorizations under the public health emergency of the coronavirus pandemic for multi-analyte respiratory panels, the US FDA clarified this week. The agency added a new item to its questions and answers (Q&As) for laboratories and manufacturers of tests for SARS-CoV-2, the virus that causes COVID-19. The new Q&A cites "the overlap in signs and symptoms between SARS-CoV-2 and other respiratory viral infections, including influenza." Multi-analyte panels for respiratory infections, noted the agency, "are useful when multiple respiratory pathogens are circulating at the same time, as is expected with the upcoming flu season." Learn More

An Indonesian firm was warned by the US FDA for failures in product testing and method validation, among other breaches of current good manufacturing practice (CGMP). In a November 2019 inspection of PT. MegaSura Mas, FDA inspectors found that some assays conducted for batch releases of an over-the-counter product were performed after the batches were released. According to the warning letter, the firm also used an assay testing method for the active ingredient in the finished product that was not a US Pharmacopeia (USP) method, nor was it validated by high-performance liquid chromatography. Learn More

Since the outset of our discovery of impurities called nitrosamines in some types of drugs more than two years ago, the U.S. FDA has undertaken a thorough investigation in an effort to protect patients. While nitrosamines are common in water and foods, nitrosamine impurities may increase the risk of cancer if people are exposed to them above acceptable levels and over long periods of time. For this reason, the discovery of unexpected nitrosamine impurities in some drug products is a serious concern, and the FDA has been working, in collaboration with regulatory counterparts around the world, to find and remove drugs with unacceptable nitrosamine impurities from the U.S. drug supply. Learn More

In its suite of post-transition guidance's released this week, the UK's Medicines and Healthcare products Regulatory Agency (MHRA) issued a dozen documents explaining how medicines licensure will work after the Brexit transition period expires on January 1st, 2021. The guidance, which largely mirrors the "no deal" scenario laid out in now-withdrawn guidance issued last year, explains a range of issues from converting centrally authorized products (CAPs) to UK marketing authorizations to how to register new packaging information for medicines after the transition date. Learn More

A second Trump administration appointee has been ousted at the FDA in the wake of the agency's botched announcement about an experimental therapy for COVID-19, which medical experts said damaged the health regulator's credibility with the public. An FDA spokesperson confirmed this week that John "Wolf" Wagner, a political appointee installed by the White House earlier this summer, is no longer heading the agency's office of external affairs. Instead, Heidi Rebello, a longtime FDA career official, has stepped into the position on an acting basis, overseeing all FDA public communications. Politico first reported the news. Learn More

The US FDA last month warned Mylan Laboratories and Acella Pharmaceuticals over good manufacturing practice (GMP) violations. The FDA's warning letter to Mylan cites the company for issues related to its cleaning procedures and handling of recovered solvents. While the warning letter to Acella Pharmaceuticals follows a three-week inspection that ran from December 2019 into January 2020 and uncovered issues with the company's NP Thyroid products that led to the company recalling 13 lots of the tablets due to super potency in May. The warning letter also cites Acella for its quality unit's failure to adequately oversee its CMO. Learn More