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    FDA Approves First New Sickle Cell Drug in Almost 20 Years

    The US Food and Drug Administration (FDA) on Friday granted approval to Emmaus Medical’s Endari (L-glutamine oral powder), the first treatment approved for patients with sickle cell disease in almost 20 years. Endari is approved for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder. Endari is the 24th new drug approved by FDA in 2017, already two more than all of 2016 . Richard Pazdur...
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    New Texas Law on Stem Cell Treatments: Showdown With FDA Coming?

    With a new Texas law now in the books to allow companies to sell unproven stem cell treatments without US Food and Drug Administration (FDA) approval, some experts wonder when FDA will step in to shut down companies and clinics exposing people to unapproved medical products. Similar to the “Right to Try” laws spreading across the US and attempting to undercut FDA’s regulation of investigational products, the Texas law , which had been brewing in some form since...
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    New Indication for Roche’s Actemra Means First FDA-Approved Drug to Treat Giant Cell Arteritis

    The US Food and Drug Administration (FDA) on Monday expanded the approved use of Roche’s Actemra (tocilizumab) to treat adults with giant cell arteritis, making it the first FDA-approved therapy for this condition. The efficacy and safety of subcutaneous (injected under the skin) Actemra for giant cell arteritis were established in a double-blind, placebo-controlled study with 251 patients with giant cell arteritis. A greater proportion of patients receiving subcuta...
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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 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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    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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    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 Looking for Two New Cellular and Gene Therapy Fellows

    • 28 March 2016
    The US Food and Drug Administration’s Division of Cellular and Gene Therapies is opening up two contract fellowship positions: one to study the potential of human pluripotent stem cells for developing gene and cell therapies, and one to further investigate mechanisms that RNA viruses use to inhibit host immune cell function. Candidates with research experience in virology, immunology, cell biology, molecular biology or gene therapy will receive the strongest consideratio...
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    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...
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    FDA Issues Guidance on Zika in Human Cell and Tissue Products

    The US Food and Drug Administration (FDA) on Tuesday announced new measures to prevent transmission of Zika virus via human cell and tissue products (HCT/Ps) by recommending a six-month deferral for at-risk donors. The virus, which has swept across Latin America and the Caribbean, is believed to be behind a spike in cases of microcephaly, a rare condition where an infant is born with an abnormally small head, and Guillain-Barré syndrome, a neurological disorder that can ...
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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...