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    Author offers advice for navigating combination products approval pathways

    With few comprehensive resources available covering combination products, RAPS’ recently released book, International Combination Products , by Jocelyn Jennings, MS, RAC (US, Drugs, Devices), fills a significant need for practical guidance on navigating the regulatory pathways for products combining multiple technologies. Prior to writing the book, “there was no place that I found that I could look to find all of the information I was interested in on different topics t...
  • ReconRecon

    Recon: ASCO 21 updates; Moderna goes for teen EUA

    Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.   In Focus: US Moderna says that its vaccine is effective for 12- to 17-year-olds and that it will seek F.D.A. authorization ( NYT ) ( NPR ) President Biden still hasn’t appointed a permanent FDA commissioner. That has big implications for upcoming drug approvals ( Fortune ) Bipartisan Legislation Introduced to Advance Biomedical Research Stalled by Pandemic ( Global Genes ) ...
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    EMA recommends bluebird bio's Skysona, seven other medicines

    The European Medicines Agency (EMA) issued a favorable opinion for bluebird bio’s Skysona (elivaldogene autotemcel), a gene therapy that treats early cerebral adrenoleukodystrophy (CALD). Seven other medicines also received positive recommendations from the agency’s Committee for Medicinal Products for Human Use (CHMP) during its May meeting.   Skysona’s status as an advanced therapy medicinal product (ATMP) means it was first assessed by EMA’s Committee for Advanced T...
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    FDA: Flexibility okay for gaps in gene therapy trials

    The US Food and Drug Administration (FDA) is not amenable to changing study endpoints or sample sizes for gene therapy clinical trials but is encouraging sponsors to fill in gaps in data collection with telemedicine and remote visits.   Wilson Bryan, director of the FDA’s Office of Tissues and Advanced Therapies (OTAT) in FDA’s Center for Biologics Evaluation and Research, told a 12 May meeting of the American Society of Gene and Cell Therapy that the pandemic is promp...
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    Euro Convergence: Mind the gap in MDR's treatment of combo products

    Navigating the re-consultation process for drug-device combination products under EU’s new Medical Device Regulation can be tricky business, said panelists during a session at Euro Convergence 2021.   Speakers at the combination products-focused session shared experiences and highlighted gaps where manufacturers still need clarity from competent authorities, who themselves are feeling the strain of implementing new regulations while they continue to sort out post-Brexi...
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    Euro Convergence: Regulators and industry seek ‘sparks’ of European change

    The opening plenary of RAPS Euro Convergence 2021 saw regulators, competent authorities, and industry representatives highlighting pressing regulatory issues for in vitro diagnostics and combination products, among other topics.   The plenary kicked off with regulators presenting “sparks,” or hot button topics intended to trigger discussion; an industry panel was then given the chance to respond to proposals from the panel of regulators and notified body representati...
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    EMA: Lentiviral thalassemia treatment on hold pending cancer investigation

    The European Medicines Agency (EMA) was notified by a gene therapy firm that the company has suspended marketing of its beta thalassemia treatment Zynteglo (betibeglogene autotemcel, bluebird bio) as a precautionary measure.   In a press release , the agency related that bluebird bio’s actions came after a recipient of the related bb1111 gene therapy for sickle cell disease developed acute myeloid leukemia (AML). Another bb1111 recipient has also developed myelodyspla...
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    FDA guides on COVID considerations in cell and gene therapy

    Manufacturers of cell and gene therapies have a new guidance from the US Food and Drug Administration (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 Infect...
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    FDA offers first thoughts on neurodegenerative disease gene therapies

    The US Food and Drug Administration (FDA) has issued draft guidance on the development, testing, and trial design for human gene therapies for neurodegenerative diseases. The document, released on 5 January 2021, 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 FDA before the submission of an investigational new drug...
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    EMA updates advanced therapy guidelines for developers

    The European Medicines Agency (EMA) has updated its guidance to industry on the development of new medicines with genetically modified cells, including a special section on considerations for developing chimeric antigen receptor (CAR) T-cell therapies.   “It is recognised that this is an area under constant development and the guideline should be applied to any novel product as appropriate,” the EMA wrote in the new guideline.   The 36-page guideline provides rec...
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    FDA finalizes feedback processes for combo products

    Developers of combination products have new final guidance from the US Food and Drug Administration 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, 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 CPA...
  • Feature ArticlesFeature Articles

    November's Regulatory Focus: US, China, and EU: Regulatory and strategic considerations

    Feature articles during November focused on a range of topics, including a comparison of US drug regulatory pathways with those in China, as well as the application of EU and US guidelines during product development for advanced therapies. The line-up also included articles on the challenges in getting contemporaneous approval for medicines and companion diagnostics, and the importance of early planning in preparation for the transition to the EU In Vitro Diagnostic Devi...