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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...
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    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 ...
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    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 ...
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    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...
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    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...
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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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    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...
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    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...
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    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...
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    September’s Regulatory Focus: Countdown to EU MDR and IVDR

    Feature articles during September focused on aspects of the EU Medical Device Regulation (MDR) and In Vitro Diagnostic Regulation (IVDR), with articles on compliance and manufacturer obligations, and perspectives examining the current status of the regulations. The issue also included the second part of the cell and gene therapy series, and an article on regulatory tools for generic drug companies.   MDR and IVDR, close up Full implementation of MDR is slated for ne...
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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...