• Regulatory NewsRegulatory News

    CAR T cell product development guidance: Comments ask for CMC details

    Both industry and clinicians asked the US Food and Drug Administration (FDA) for clarification related to the evaluation of cellular starting materials in its draft guidance on chimeric antigen receptor (CAR) T cell product development.   The public comments also sought more details on change management and how the guidance applies to other genetically modified products.   The draft guidance , issued on 15 March 2022, provides recommendations on chemistry, manufac...
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    Marks eyes gene therapy development pilot, sees uptick in successful RMAT requests

    Peter Marks, director of Center for Biologics Evaluation and Research (CBER) at the US Food and Drug Administration (FDA), attributes an increase in the amount of regenerative medicine advanced therapy (RMAT) requests approved for cell and gene therapies to sponsors “getting the hang” of the agency’s expectations for designation requests.   Marks spoke at the California Separation Science Society’s (CASSS) meeting on 8 June on cell and gene therapies. The meeting was h...
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    FDA explains plans to bolster cell and gene therapy approvals through wider messaging

    Updated 25 May 2022 to correct ASGCT's name. Wilson Bryan director of the US Food and Drug Administration’s Office of Tissues and Advanced Therapies (OTA) in the agency’s Center for Biologics Evaluation and Research, told a 19 May meeting  of the American Society of Gene and Cell Therapy (ASGCT) that the division is undertaking a series of internal measures to improve communications with sponsors and to widen its messaging.   These measures are being taken to com...
  • RF Quarterly

    Digital therapeutics: Leveraging the SaMD framework for regulatory success

    The digital health sector is experiencing enormous growth globally. Briefly, digital health is a branch of healthcare that is primarily focused on improving health using internet, digital, and mobile technologies. One such novel subclass of the digital health ecosystem is digital therapeutics (DTx). DTx are “software-driven,” nonpharmacological therapeutic interventions that can improve the accessibility and effectiveness of healthcare. This article explores the current di...
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    Stakeholders seek clarity on FDA cell and gene therapy draft guidance

    Industry, medical societies and other stakeholders have weighed in on draft guidance from the US Food and Drug Administration (FDA) detailing how sponsors that want to study multiple versions of cell and gene therapies could combine them in a single “umbrella” trial.   The draft guidance, released in September 2021, outlined FDA’s proposed thinking for how this process would work. For cell and gene therapies intended to treat a single disease, sponsors would typically ...
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    Gene therapy developers can expect less hand-holding from OTAT

    A growing workload and insufficient staffing have prompted the US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) to institute new processes to expedite its reviews of new cell and gene therapies, said OTAT Director Wilson Bryan at a 6 December webinar sponsored by the Alliance for a Stronger FDA.   At the webinar, Bryan was asked to discuss the impact of the projected workload for OTAT, projections for approving new cell and gene t...
  • Feature ArticlesFeature Articles

    The regulatory landscape of advanced therapies in Asia-Pacific

    The Asia-Pacific region, which inlcudes Australia, China, Hong Kong, Japan, Malaysia, Singapore, and South Korea, has a significant number of clinical trials in advanced therapies, with China ranking second globally. Manufacturers in other countries in the region, such as Japan, Australia, South Korea, and Singapore, have submitted marketing authorizations applications for advanced therapies and received approvals. Regulators recognize this emerging trend in dynamic resear...
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    For gene therapies, FDA drafts trial guidance, finalizes "sameness" for orphan exclusivity

      A new draft guidance from the US Food and Drug Administration (FDA) gives cell and gene therapy developers a detailed framework for the conduct of umbrella trials, offering the potential for enhanced speed and efficiency in early-stage clinical trials.   In the umbrella trials envisioned under the draft guidance, two or more versions of a cellular or gene therapy product would be studied for one specific disease using just one trial design, shared infrastructure, a...
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    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 ...
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    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

    Recent ruling gives FDA ammunition against unapproved cell therapies

    A recent appeals court decision strengthens the US Food and Drug Administration’s efforts to crack down on clinics making unapproved stem cell therapies, the agency’s Melissa J. Mendoza said at a recent meeting.   The 11 th US Circuit Court of Appeals rendered a decision in USA vs US Stem Cell Clinic LLC , agreeing with the “FDA that these products did not fit the same surgical procedure exemption” and should be regulated as drugs, said Mendoza, deputy director of ...