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  • Regulatory NewsRegulatory News

    Convergence: EMA close to finalizing guidance for advanced therapies

    The European Medicines Agency is on the verge of releasing revised guidance for advanced therapy medicinal products containing genetically modified cells, which includes chimeric antigen receptor (CAR)-T cell therapies. The “Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells” was originally issued in 2012 but underwent revision and consultation from July 2018-July 2019. The revised version is expected to...
  • Regulatory NewsRegulatory News

    House Reps Seek More Transparency on Novartis-CMS Pricing Deal for Newly Approved CAR-T Therapy

    Rep. Lloyd Doggett (D-TX) and other House Democrats on Wednesday sent a letter to Seema Verma, administrator of the Centers for Medicare & Medicaid Services (CMS), seeking to find out more information about an outcomes-based payment approach for Novartis’ newly approved chimeric antigen receptor T-cell (CAR-T) treatment Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). The outcomes-based pay...
  • 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...
  • Regulatory NewsRegulatory News

    Nature Editorial: FDA Needs Help Assessing Cutting-Edge Tech

    • 07 August 2017
    The US Food and Drug Administration (FDA) is increasingly being tasked with evaluating cutting-edge therapies and technology that its in-house expertise may not be able to address, according to a Nature editorial, which recommends academic scientists help the agency. Using the example of the 12 July meeting of the Oncologic Drugs Advisory Committee that voted unanimously in favor of the benefit-risk profile for the first of a new kind of cancer therapy, the ed...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    CBER Director Offers a Peek Into the Complexities of Where the Center is Headed

    In addition to protecting the US blood supply and ensuring vaccines are safe and effective, the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) is also now regulating complex gene and cell therapies, as well as a new category of treatments: regenerative medicines. Peter Marks, director of CBER, offered attendees of DIA’s annual conference on Tuesday a few examples of the changing landscape for the center, noting that with...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    FDA Proposes New Databases to Monitor CAR T-Cell Safety Across INDs

    The US Food and Drug Administration (FDA) wants to create two new databases that will allow it to look at safety and manufacturing information across multiple applications for products within a promising class of cancer immunotherapies called anti-CD19 CAR modified T-cells. FDA officials presented their proposal to pilot the databases last week during a meeting of the Recombinant DNA Advisory Committee (RAC), which is organized by the National Institutes of Health's (NIH...
  • EMA Initiates Review of Low-T Medications

    The European Medicines Agency's (EMA) has announced it will initiate a review of testosterone-containing medicines following concerns that the drugs, used primarily to treat a condition commonly known as "Low-T," may cause an elevated risk of cardiovascular side effects. Background Those concerns were first raised by the US Food and Drug Administration (FDA) in February 2014 following the publication of two separate studies in the Journal of the American Medical Asso...
  • Endo's Aveed Approved by FDA, Drawing Anger from Consumer Group

    US regulatory officials today approved Endo Pharmaceuticals' testosterone replacement therapy drug Aveed (testosterone undecanoate), ignoring a petition submitted last week by a consumer group that claimed the agency should first conclude a safety review of alternative products. Background FDA recently initiated a safety review of testosterone replacement therapy drugs-more commonly known as "Low-T" drugs-after several studies raised concerns about their cardiovascular ...
  • Consumer Group Calls for New Warnings for Low-T Products, Delay of Endo's Aveed

    Less than a month after US regulators issued a preliminary warning about the cardiovascular risks associated with products intended to treat low testosterone levels in men, a prominent consumer advocacy group is calling on the US Food and Drug Administration (FDA) to add more serious warnings to the products. Background FDA's review of the products, commonly known as "low-T" drugs, was triggered by two separate studies. The studies, in the  Journal of the American...