• Regulatory NewsRegulatory News

    EMA, European Commission Look to Foster Development of Advanced Therapies

    The European Medicines Agency (EMA) and European Commission on Friday published a joint action plan to further the development of new cell and gene therapies, as well as other advanced therapy medicinal products (ATMPs). The plan, which features the release of several new and revised guidelines comes as on the international level, a regular forum for dialogue has been established to share experiences on advanced therapies between EMA, the US Food and Drug Administr...
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    FDA Panel Votes Unanimously in Favor of Gene Therapy to Treat Rare Eye Disorder

    Philadelphia-based Spark Therapeutics took its gene therapy before the US Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee on Thursday and the panel voted 16 to 0 in favor of approving the subretinal injection, representing a major step forward for adeno-associated virus gene therapies. The therapy, known as Luxturna (voretigene neparvovec), has been granted priority review by FDA (the agency has until 12 January 2018 t...
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    'Historic Action': FDA Approves First CAR-T Therapy

    The US Food and Drug Administration (FDA) on Wednesday approved the first gene therapy, Novartis' Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). Dubbed an "historic action" by the agency, the approval comes a little more than a month after FDA's Oncologic Drugs Advisory Committee voted unanimously , 10 to 0, in favor of the benefit-risk profile for the first of this new kind of cancer th...
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    FDA to Unveil New Regulatory Framework for Stem Cell Therapies

    The US Food and Drug Administration's (FDA) Commissioner Scott Gottlieb said Monday the agency will advance a new framework to better regulate stem cell therapies this fall. "This comprehensive policy will establish clearer lines around when these regenerative medicine products have sufficient complexity to fall under the agency's current authority, and then define an efficient process for how these products should be evaluated for safety and effectiveness," Gottli...
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    Do Drugs Approved Via Expedited Pathways Offer Greater Benefits? Study and Doctors Debate

    A recent study in Health Affairs suggests that drugs given an expedited review by the US Food and Drug Administration (FDA) offer greater health gains than drugs that receiving a conventional review. But experts caution that the study might only show incremental benefits. The study, conducted by Peter Neumann, director of the Center for the Evaluation of Value and Risk in Health and three of his colleagues at Tufts Medical Center, compared the change in quality-adjuste...
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    FDA: US Clinical Research Using Three-Parent Baby Technique is Illegal

    The US Food and Drug Administration (FDA) said Friday that it is illegal to perform research in the US on a new type of technology that showed promise last year in helping a woman who had multiple miscarriages because of a genetic disorder to give birth to a baby boy in Mexico without that disorder. The technology, known as mitochondrial replacement technology (MRT), deals with mitochondrial DNA passed down only from mother to child. In September 2016, MRT was used to...
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    FDA Panel Votes Unanimously in Favor of First CAR-T Cancer Therapy

    The US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee on Wednesday voted unanimously, 10 to 0, in favor of the benefit-risk profile for the first of a new kind of cancer therapy, known as a Chimeric Antigen Receptor T-cell (CAR-T) therapy. Although the outside panel of experts raised questions about concerns with the safety and manufacturing of the Novartis treatment, known as CTL019 (tisagenlecleucel-T), the panel did not question the effi...
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    CBER Director Focuses on Flexibility to Advance Regenerative Medicines

    Peter Marks, director of the US Food and Drug Administration's (FDA) Center for Biologics Evaluation and Research (CBER) on Thursday said his office is equipped to support the development of cutting edge treatments, such as cell and gene therapies. Speaking at the Food and Drug Law Institute's annual conference, Marks said the 21st Century Cures Act added tools to FDA's regulatory arsenal, including the regenerative medicine advanced therapy (RMAT) designation to provi...
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    FDA Officials Question Lack of New Cancer Drugs Developed in Combination With Radiation

    Although almost half of patients with cancer who are cured will have received radiation therapy as a component of their care, there is still a dearth of new therapies being developed in combination with radiation, officials from the US Food and Drug Administration (FDA) wrote in an article published this week. The authors, including Richard Pazdur, director of FDA's Oncology Center of Excellence, and FDA medical officer Amanda Walker, say that since 2006 there have been ...
  • Feature ArticlesFeature Articles

    Regulation and Oversight of Gene Therapy in the US

    This article discusses the evolution, potential uses and regulatory aspects of gene therapy and includes funding, delivery mechanisms, bioethics and steps toward clinical trial implementation. Introduction Sponsors and investigators need to be prepared to take specific steps to satisfy FDA and NIH requirements before, during and after clinical trial implementation. There are a significant number of guidance documents and internal procedures aimed at helping to prepare...
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    FDA Begins Accepting Regenerative Therapy Applications for RAT Designation

    The US Food and Drug Administration (FDA) on Thursday unveiled a new designation category created as part of the 21st Century Cures Act to allow drugs to be eligible for designation as a regenerative advanced therapy (RAT). The stipulations for such a RAT designation include: “The drug is a regenerative medicine therapy, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such the...
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    FDA Withdraws Proposed Rule on Public Disclosure of Info on Unapproved Gene Therapies

    The US Food and Drug Administration (FDA) on Thursday announced it is withdrawing a proposed rule from 2001 that would have required the public disclosure of summaries of safety and effectiveness data, in addition to other information, from pre-market clinical trials of gene therapies and transplanted non-human tissues to humans, known as xenotransplantation. “FDA has reconsidered our position on this issue and deemed our concerns from 2001 outdated. We will continue to ...