• RoundupsRoundups

    Asia-Pacific Roundup: TGA releases risk classification guidance for active medical devices

    TGA releases guide to classifying active medical devices by risk   New guidance from Australia’s Therapeutic Goods Administration (TGA) on the risk classification of active medical devices covers software-based products and other medical devices that act by converting energy. Examples of covered devices include pacemakers and phototherapy devices.   The EU defined the term “active medical device” in its Medical Device Regulation (MDR). TGA, which follows the EU w...
  • Regulatory NewsRegulatory News

    Convergence: Inadequate testing of cell and gene therapies draws FDA concern

    The lack of consistent testing in measuring product quality for cell and gene therapies is a major deficiency found by US Food and Drug Administration (FDA) reviewers in its information requests (IRs) to manufacturers in their submission of biologics license applications (BLAs).   This observation was made by Kedest Tadesse, senior research manager for Agency IQ, which recently compiled a survey of deficiencies identified in IR letters in FDA’s review of five approved ...
  • 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...
  • Feature ArticlesFeature Articles

    Regulation of cell and gene therapy products in Japan

    In recent years, new cell and gene therapies have been developed and approved in a number of countries, including Japan. The Japanese government fully supports academia and the pharmaceutical industry in the research and development of these innovative therapies, and in 2019, revised the Act on Securing Quality, Efficacy, and Safety of Products Including Pharmaceuticals and Medical Devices to enact the conditional early approval system for accelerating the development and ...
  • Regulatory NewsRegulatory News

    Cures 2.0: Discussion draft signals impact on FDA, creation of ARPA-H

    More than four years after the 21 st Century Cures Act was signed into law, Reps. Diana DeGette (D-CO) and Fred Upton (R-MI) on Tuesday circulated a discussion draft of their proposed Cures 2.0 legislation, setting the stage for negotiations on the long-awaited legislative package.   In addition to proposing new programs and enhancements for the US Food and Drug Administration (FDA) and the Centers for Medicare and Medicaid Services (CMS), the 127-page draft bill ...
  • Regulatory NewsRegulatory News

    Euro Convergence: Academic clinics should establish risk management programs for ATMP development

    Academic centers should establish risk management programs to successfully commercialize cell and gene therapy products and to be a viable partner with contract research organizations (CROs) and manufacturers in developing these therapies.   So asserted Lutz Uharek, a professor of internal medicine and founder and CEO of Xencura, at RAPS Euro Convergence 2021 on 12 May in discussing some of the challenges and opportunities for academic centers in developing cell and ge...
  • Regulatory NewsRegulatory News

    New laws address NCE exclusivity, biosimilar education

    Two bipartisan bills aimed at clarifying what constitutes a new chemical entity (NCE) and providing education on biosimilars to consumers and physicians were signed into law by President Joe Biden on Friday.   The first bill, S. 415 , also known as the Ensuring Innovation Act , amends the Federal Food, Drug, Cosmetic Act (FD&C Act) to replace the definition of an active ingredient with the active moiety definition used by FDA for the purposes of determining whether...
  • Regulatory NewsRegulatory News

    Drugmakers suggest tweaks to FDA’s neurodegenerative disease gene therapy guidance

    Several drugmakers and Health Canada are suggesting changes to the US Food and Drug Administration’s (FDA) recently released draft guidance on developing gene therapies for neurodegenerative diseases.   Released in January, the draft guidance provides recommendations for the development, testing and clinical study of gene therapies for neurodegenerative diseases. The guidance also discusses considerations for chemistry, manufacturing and controls (CMC) and offers an ov...
  • Regulatory NewsRegulatory News

    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...
  • 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...
  • Regulatory NewsRegulatory News

    Marks fields questions on cell and gene therapy development

    Expect new guidance from the US Food and Drug Administration (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 agen...
  • Feature ArticlesFeature Articles

    Advanced therapies: Navigation and application of EU and US guidelines during product development

    Continued progression and understanding in the development of advanced therapies has led to a significant increase in the number of products and types of indication under investigation, particularly for treating serious and life-threatening conditions. Although the principle regulatory requirements and guidance, as issued for traditional biologic products, can be generally applied, advanced therapies require additional regulatory and strategic considerations because of the...