• Regulatory NewsRegulatory News

    FDA elevates OTAT to “Super Office” within CBER

    The US Food and Drug Administration (FDA) announced this week it has elevated and reorganized its Office of Tissues and Advanced Therapies (OTAT) to a “Super Office” within the Center of Biologics Research and Evaluation (CBER) to meet its growing workload and new commitments under the Prescription Drug User Fee Act (PDUFA VII) agreement for FY2023-2027.   The office will be renamed the Office of Therapeutic Products (OTP).   “With the current and anticipated incre...
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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...
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    Stakeholders seek clarity on FDA cell and gene therapy draft guidance

    Industry, medical societies and other stakeholders have weighed in on draft guidance from the US Food and Drug Administration (FDA) detailing how sponsors that want to study multiple versions of cell and gene therapies could combine them in a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   The draft guidance, released in September 2021, outlined FDA’s proposed thinking for how this process would work.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...
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    FDA rolls out more guidance on 'N of 1' gene therapies

    Developers of individualized investigational antisense oligonucleotide (ASO) therapies for ultra-rare diseases received additional guidance from the US Food and Drug Administration (FDA). In two draft documents, the agency has provided new information for sponsor-investigators and for those overseeing manufacture of these so-called “N of 1” therapies for people with severely debilitating or life-threatening genetic disease.   "Progress in individualized medicines provi...
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    New public-private consortium will tackle gene therapies for rare diseases

    Two federal agencies are partnering with pharmaceutical companies and non-profit organizations to form a consortium aimed at boosting the development of gene therapies for rare diseases.   In a Wednesday announcement, the US Food and Drug Administration (FDA) and the National Institutes of Health (NIH) revealed the launch of the Bespoke Gene Therapy Consortium (BGTC). The consortium’s work will be managed by the Foundation for the NIH and sit within the NIH Acceleratin...
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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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    Convergence: Regulatory considerations in advancing gene and cellular therapies

    When developing a regulatory submission for a gene or cellular therapy, it’s essential to keep the basics in mind, such as how to demonstrate the product’s identity, purity, and potency, Haroon Hashmi, PhD, told attendees at RAPS Convergence 2021.   “Even when we think about these complex technologies -- new emerging technologies -- certain regulatory principles always stay in place,” said Hashmi, senior vice president at Ziopharm Oncology, which has developed the Slee...
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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 ...