• 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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    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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    Regulation of cell and gene therapy products in China

    This article provides an overview of the Chinese regulations and guidance documents for cell and gene therapy products (CGTPs).   Introduction As early as 1993, Chinese regulators provided guidance on clinical research for somatic cell and gene therapy. More regulations, laws, and guidances have since been published, either specific to, or including, CGTPs. The two most recent publications have been draft guidelines for clinical studies for immune cell therapy produc...
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    Gene therapies – The regulatory road to individualized medicine

    This article discusses the evolving US regulatory landscape for gene therapies. The author explores the recent history and developmental paradigm shifts for gene therapy products and concludes with advice for abbreviated development and thoughts on policies that will create a sustainable gene therapy ecosystem. The author examines the paradigm shift of clinical trial and lifecycle management for gene therapies and considers the changes to the US regulatory framework that ...
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    Gene therapies: Industry asks for clarification on FDA’s sameness guidance

    Biotech companies and industry groups are raising questions about the US Food and Drug Administration’s (FDA) recent draft guidance on interpreting sameness of gene therapy products under its orphan drug regulations.   The draft guidance, issued for comment in January, explains how FDA intends to determine the sameness of two gene therapies intended for the same use or indication for the purposes of awarding orphan drug designation and exclusivity. (RELATED: FDA fina...
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    Regulation of advanced therapy medicinal products in the EU

    This article explains some of the terminology relating to advanced therapy medicinal products (ATMPs), including gene and cell-based therapies, tissue-engineered products, and combined ATMPs. The author explains the key EU regulations and guidance documents for each therapy type and discusses marketing authorization, accelerated regulatory pathways, and market access. He advises companies navigating this complex regulatory environment to engage with the regulatory agencies...
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    Cell and gene therapies: FDA official on COVID-19 impact

    Speaking at a session on cell and gene therapies at BIO Digital on Monday, a top US Food and Drug Administration (FDA) official discussed the impact of the coronavirus disease (COVID-19) pandemic on the agency’s work related to cell and gene therapies.   “Before the pandemic, we were seeing a huge escalation in the workload that we have to make adjustments for,” said Wilson Bryan, director of the Office of Tissues and Advanced Therapies at the Center for Biologics Eval...
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    Bespoke therapies – opportunities, challenges, and hope

    This article discusses the advent of bespoke therapies, defined as the tailoring of medical treatment to the individual characteristics or symptoms and responses of a patient during all stages of care and as a new frontier beyond personalized medicine. The author covers the revolutionary genetic tools implementing such therapies and the clinical and nonclinical safety perspectives for bespoke therapies. The author concludes that with bespoke therapies we are entering a new...