• Regulatory NewsRegulatory News

    '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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    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...
  • Feature ArticlesFeature Articles

    Advanced Therapy Medicinal Products: An Evolving Regulatory Landscape

    This article was developed from a presentation at RAPS Regulatory Convergence in September 2016 in San Jose, California, US. It was the opening presentation in the "Global Developments in Cell and Gene Therapy" session and covered introductory Advanced Therapy Medicinal Product (ATMP) concepts from definitions to strategic considerations and challenges currently faced by developers in this field. Introduction Broadly speaking, Advanced Therapy Medicinal Products (ATM...
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    Microbial Vectors Used for Gene Therapy: FDA Offers Final Guidance

    The US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research this week issued final guidance for investigation new drug (IND) submissions for microbial vectors used for gene therapies (MVGTs) in early-phase clinical trials. The 27-page final guidance follows a draft issued in October 2015 and is meant to supplement: “Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human...
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    Cancer Immunotherapies: EMA to Hold November Workshop

    The European Medicines Agency (EMA) on Tuesday announced that its Committee for Advanced Therapies (CAT) is organizing a workshop on 15 and 16 November 2016 to discuss the scientific and regulatory challenges of immunotherapy medicines based on genetically modified T-cells (white blood cells that fight off viruses and bacteria). As EMA explains, “Immunotherapy is a type of cancer treatment that mobilizes the body's own defense mechanisms to fight cancer. Immunotherapy m...
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    EC Report Analyzes Regulatory Frameworks for Advanced Therapies in US, Canada, Japan and Korea

    The European Commission report released Wednesday dissects the similarities in how gene-, cell- and tissue-based advanced therapies are regulated across the four different geographic regions, though ongoing research projects in such therapies are heavily concentrated in the US and Japan. The 327-page report highlights the “high degree of convergence” in the regulation of advanced therapies across the US, Canada, South Korea and Japan, noting that only Japan has enacted (...
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    Advanced Therapies: Stakeholders Call for EMA Changes on Regulations, Manufacturing

    The European Medicines Agency (EMA) on Friday released a report outlining the concerns and regulatory changes described by developers, manufacturers and investors in advanced therapy medicinal products (ATMPs), which include gene therapies, tissue-engineered products and somatic cell therapies, at a meeting last week. The report notes that recurring themes included the need for early interaction with regulators and guidance, more transparency, greater harmonization betwe...
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    EMA Unveils 19 Advanced Therapy Product Classifications

    The European Medicines Agency (EMA) on Tuesday released a host of classification decisions for advanced therapies being developed to treat various types of tissue damage, including burns, scars and non-healing wounds. The agency, through its Committee for Advanced Therapies (CAT), offers sponsors an optional consultation to determine whether a product meets the criteria for an advanced-therapy medicinal product (ATMP) . The classification procedure is laid out in Arti...
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    FDA Sees Spike in Gene and Cell Therapy Applications

    The US Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) met Tuesday to discuss updates of research programs in the Office of Cellular, Tissue and Gene Therapy (CTGT), which is part of FDA’s Center for Biologics Evaluation and Research (CBER). The research updates came as Raj Puri, MD, PhD, Director, Division of Cellular, and Gene Therapies (DCGT) at CBER told the committee that although his division has only licensed 12 prod...
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    Senate Bill Seeks to Create Standards Body for Regenerative Medicine

    Sen. Tammy Baldwin (D-WI) introduced a bill on Tuesday to promote the development of regulatory science and standards for regenerative medicine and advanced therapies. The bill, known as the Advancing Standards in Regenerative Medicine Act , would require the US Food and Drug Administration (FDA) to oversee the creation of a "Standards Coordinating Body" for regenerative medicine. Regenerative medicine and advanced therapies belong to a growing field of medicine that i...
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    FDA Authorizes First Human Study to Use In Vivo Genome Editing Application

    The US Food and Drug Administration (FDA) broke new ground last week when it authorized the world’s first human clinical trial for an  in vivo  genome editing application. The investigational new drug (IND) application approval for California-based Sangamo, which is the first and only company to put a gene editing therapy into clinical trials, allows the company to begin conducting in 2016 a Phase 1/2 open-label, dose-escalation study in up to nine male adults with sever...
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    Cancer Gene Therapies: FDA Officials Highlight Regulatory Approaches

    As gene therapies begin to get a foothold among other cancer treatments, the US Food and Drug Administration (FDA) is taking a more flexible, data-driven approach for the preclinical testing programs of these biologically complex products, according to a new review from five FDA officials in Cancer Gene Therapy . Although FDA has yet to approve a gene therapy to treat cancer, the authors note that about two-thirds of gene therapy clinical trials are for cancer treatment...