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  • Regulatory NewsRegulatory News

    This Week at FDA: New proposed rules, Biocon's Form 483, and more

    We're trying something new at Regulatory Focus , with the goal of gathering the week’s news from (and about) FDA. We'll be covering agency activities and enforcement actions, filings in the Federal Register , new guidances, legislative updates, and anything else we come across – all in one place. We hope you will find it to be your one-stop-shop for FDA updates. What do you think? We’d be happy to hear your feedback at news@raps.org. News from this week One of t...
  • Feature ArticlesFeature Articles

    US regulations for regenerative medicine advanced therapies

    This article examines US regulations and guidance documents for regenerative medicine advanced therapies (RMATs). The author describes the field of regenerative medicine, noting that it is expanding at an accelerated pace, and outlines some of the common terms associated with it. He also addresses the application process for these therapies, accelerated regulatory pathways, market access, and the outlook for RMATs. The author cautions that, as exciting as these therapies a...
  • Feature ArticlesFeature Articles

    Update on RMAT designations

    This article discusses the scope and purpose of the special designation for regenerative medicine advanced therapies (RMAT) created by the passage of the 21st Century Cures Act. The authors explain the benefits expected to be realized with RMAT and provide a tally of products receiving the special designation to date and a current count, by year, of products for which RMAT designation has been requested.   Introduction Section 3033 of the 21st Century Cures Act, titl...
  • Regulatory NewsRegulatory News

    Two Gene Therapy Approvals Headline CBER’s FY 2018 Report

    The US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) on Wednesday published its Fiscal Year 2018 report, highlighting two gene therapy approvals, as well as the approvals of a hepatitis B vaccine, a vaccine to prevent shingles and a flu vaccine for children as young as six months. Both of the gene therapy approvals occurred at the very beginning of the previous fiscal year, with CBER signing off on Spark Therapeutics’ Luxturna...
  • Regulatory NewsRegulatory News

    FDA Unveils New Regenerative Medicine Framework

    The US Food and Drug Administration (FDA) on Thursday launched a new policy framework for regenerative medicine, building off a previous framework from 2005, as part of efforts to bring new cell, stem cell and tissue products to patients as efficiently as possible while managing the proliferation of unscrupulous actors hawking unproven therapies. FDA’s announcement included the release of two new draft guidance documents – one on ways to expedite approvals for regen...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    Humacyte Receives First of FDA’s Regenerative Medicine Designations

    The US Food and Drug Administration (FDA) on Monday offered one of its first Regenerative Medicine Advanced Therapy (RMAT) designations to Humacyte’s investigational human acellular vessel (HAV) Humacyl. “Being one of the first companies to receive the Regenerative Medicine Advanced Therapy Designation from the FDA is an honor and a testament to the significant potential for Humacyl to address a great unmet medical need for patients who are undergoing dialysis,” Ca...