• Regulatory NewsRegulatory News

    Animal models have limitations for safety assessment of gene therapies: FDA adcomm

    An advisory committee to the US Food and Drug Administration (FDA) has said animal models are “problematic” in assessing the safety risks of gene therapies derived from adeno-associated virus (AAV) vectors.   FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) held a virtual meeting on 2 September to discuss the best animal models to study to assess safety of these products.   “AAV are a popular type of gene therapy, yet we are seeing a variety o...
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    Chisholm, Falcone, Soo and Steffen named RAPS Fellows

    RAPS today announced the names of four exceptional senior regulatory professionals who will join the venerated ranks of RAPS Fellows . The 2021 RAPS Fellows are:   Orin Chisholm , FRAPS, BSc (Hons), GCULT, PhD, SFHEA, principal consultant, Pharmed, Sydney, Australia Robert Falcone , FRAPS, PhD, senior manager, regulatory affairs, Prestige Consumer Healthcare Inc., Tarrytown, NY Chin-Wei Soo , FRAPS, DRSc, global regulatory head, devices and combination products,...
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    JAK inhibitors' CV, cancer risks merit expanded boxed warning

    The boxed warnings for several members of a class of drugs used to treat a number of inflammatory conditions must be revised to reflect the increased risk of cancer, serious cardiovascular problems and death seen in people taking the drugs, according to a new drug safety communication from the US Food and Drug Administration (FDA).   The revisions come after safety data from a randomized clinical trial of Pfizer’s Xeljanz (tofacitinib) and is extended release formulati...
  • RoundupsRoundups

    Euro Roundup: Commission seeks feedback on proposed EudraVigilance changes

    The European Commission is seeking input on planned changes to pharmacovigilance regulations. Based on feedback from the European Medicines Agency (EMA), the Commission is proposing to add rules about EudraVigilance monitoring and contracts with pharmacovigilance service providers.   Overall, the Commission said the experience with the pharmacovigilance implementing regulation is “good.” However, talks with EMA and the Pharmacovigilance Risk Assessment Committee have i...
  • ReconRecon

    Recon: FDA approves BeiGene's Brukinsa for rare blood cancer; House Committees request records from FDA's Aduhelm review

    Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.   In Focus: US Moderna seeks US authorization for COVID-19 vaccine booster ( Reuters ) House Committees Demand FDA Records on Alzheimer’s Drug Approval ( NYTimes ) Purdue Pharma Is Dissolved and Sacklers Pay $4.5 Billion to Settle Opioid Claims ( NYTimes ) ( STAT ) ( Reuters ) FDA warns of heart-related events, cancer, blood clots, and death for JAK inhibitors ( Endpoints 1 ...
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    Top FDA and EMA officials to kick off Convergence 2021

    RAPS will hold its annual Convergence, the world’s largest conference dedicated to healthcare product regulation and regulatory issues, 12–15 September . Convergence 2021 will be a live, virtual event featuring more than 100 educational sessions, presentations, panel discussions, speaker and author meet and greets, and other interactive sessions with more than 200 expert speakers.   The four-day conference will kick off with an opening plenary and panel discussion o...
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    FDA convenes adcomm for Pfizer COVID booster

    The US Food and Drug Administration (FDA) announced late Wednesday afternoon that it plans to hold an advisory committee meeting on 17 September to discuss a third booster shot of the Pfizer-BioNTech COVID-19 vaccine for individuals aged 16 years and up.   "The FDA is evaluating data submitted by Pfizer-BioNTech in a supplemental Biologics License Application for its COVID-19 vaccine and will discuss it with the agency's advisory committee to inform our decision-making...
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    FDA proposes to refuse Intarcia’s exenatide combo product NDA —again

    The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) has told Intarcia Therapeutics it is proposing to refuse a new drug application for their type 2 diabetes drug-device combination product ITCA 650.   In a notice posted in the Federal Register , the agency said Intarcia can request a hearing from FDA on the proposal, but if the hearing is waived or it is determined the reason for the hearing isn’t sufficient, FDA will move forwa...
  • RoundupsRoundups

    FDA Approvals Roundup: Skytrofa, Invega Hafyera, Briviact

    A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA). New approvals Skytrofa approved as weekly therapy for children with growth hormone deficiency Ascendis’s  Skytrofa (lonapegsomatropin-tcgd injection) has been approved as a weekly human growth hormone treatment for children aged 1 year or older who have short stature because of growth hormone deficiency.   The approval was based on efficacy findings from a...
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    FDA revises MAPPs on data standards program, scientific interest groups

    The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) recently issued two revised documents for its Manual of Policies and Procedures (MAPP): one addresses updated procedures for its data standards program and the other covers operational procedures for scientific interest groups (SIG).   The 10-page MAPP on data standards governance discusses the operating model to support the implementation of CDER’s data standards program. It r...
  • ReconRecon

    Recon: Novartis' Leqvio nabs NICE nod; HebeCell makes a foray into CAR-NK space

    Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.   In Focus: US When Will the Delta Surge End? ( NYT ) Why can’t America fix its Covid-19 testing problems? ( VOX ) Vaccination Rates Rose In August As COVID Cases Surged Due To The Delta Variant ( NPR ) First vaccination doses up 17 percent since Pfizer approval: analysis ( The Hill ) Companies eye financial penalties for unvaccinated workers ( The Hill ) America has waste...
  • Feature ArticlesFeature Articles

    Emergency use authorizations for IVDs: Building on lessons learned

    Emergency use authorization (EUA) is a tool used by the US Food and Drug Administration (FDA) for providing quick access to unapproved medical products in response to a public health emergency. In vitro diagnostics (IVDs) have been critical EUA products in past emergencies and have proven equally important during the COVID-19 pandemic. For regulatory professionals, the valuable lessons learned from EUA policy and ensuing changes over time have provided a crucial foundation...