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  • Regulatory NewsRegulatory News

    Combination products: FDA releases PDUFA VI program report

    A new independent report on the US Food and Drug Administration’s (FDA) review practices for combination products under the Prescription Drug User Fee Act (PDUFA VI) gives the agency generally positive marks but finds room for refinements in communication and technology to improve the pre-submission and review process.   The report , commissioned by FDA as part of its PDUFA VI commitments, was conducted by Eastern Research Group and looked at FDA staff and sponsor e...
  • Regulatory NewsRegulatory News

    Gene therapies: Industry asks for clarification on FDA’s sameness guidance

    Biotech companies and industry groups are raising questions about the US Food and Drug Administration’s (FDA) recent draft guidance on interpreting sameness of gene therapy products under its orphan drug regulations.   The draft guidance, issued for comment in January, explains how FDA intends to determine the sameness of two gene therapies intended for the same use or indication for the purposes of awarding orphan drug designation and exclusivity. (RELATED: FDA fina...
  • Feature ArticlesFeature Articles

    Regulation of advanced therapy medicinal products in the EU

    This article explains some of the terminology relating to advanced therapy medicinal products (ATMPs), including gene and cell-based therapies, tissue-engineered products, and combined ATMPs. The author explains the key EU regulations and guidance documents for each therapy type and discusses marketing authorization, accelerated regulatory pathways, and market access. He advises companies navigating this complex regulatory environment to engage with the regulatory agencies...
  • 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

    Cell and gene therapies: FDA official on COVID-19 impact

    Speaking at a session on cell and gene therapies at BIO Digital on Monday, a top US Food and Drug Administration (FDA) official discussed the impact of the coronavirus disease (COVID-19) pandemic on the agency’s work related to cell and gene therapies.   “Before the pandemic, we were seeing a huge escalation in the workload that we have to make adjustments for,” said Wilson Bryan, director of the Office of Tissues and Advanced Therapies at the Center for Biologics Eval...
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    FDA releases compliance program for CDER, CDRH-led combination product inspections

    In a newly issued compliance program , the US Food and Drug Administration (FDA) explains its approach to inspections of Center for Drug Evaluation and Research (CDER)- and Center for Devices and Radiological Health (CDRH)-led combination products.   The 46-page document lays out FDA’s intended implementation of the compliance program; inspectional operations and reporting considerations; sampling and analytical testing expectations; regulatory and administrative stra...
  • Feature ArticlesFeature Articles

    Bespoke therapies – opportunities, challenges, and hope

    This article discusses the advent of bespoke therapies, defined as the tailoring of medical treatment to the individual characteristics or symptoms and responses of a patient during all stages of care and as a new frontier beyond personalized medicine. The author covers the revolutionary genetic tools implementing such therapies and the clinical and nonclinical safety perspectives for bespoke therapies. The author concludes that with bespoke therapies we are entering a new...
  • Regulatory NewsRegulatory News

    Refine gene therapy follow-up guidance, expert says

    It’s time for regulatory agencies, academics and pharmaceutical companies to convene to refine guidance for long-term follow-up of patients receiving gene therapy. That was the message Anne-Virginie Eggimann, senior vice president for regulatory science at bluebird bio, Inc., brought to a policy session at the annual meeting of the American Society of Gene & Cell Therapy .   To date, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) hav...
  • Regulatory NewsRegulatory News

    FDA abandons proposal for devices referencing drugs

    The US Food and Drug Administration (FDA) on Thursday said it will abandon a proposed regulatory approach for medical devices referencing drugs (DRDs) that would have allowed devices to be authorized for new uses with already approved drugs when the drugmaker does not want to collaborate on the new use.   The decision comes after FDA held a public hearing to discuss the scientific, regulatory and legal challenges posed by the approach in November 2017. At the time, FDA...
  • Regulatory NewsRegulatory News

    FDA drafts guidance on emergency-use injector reliability

    The US Food and Drug Administration (FDA) this week issued draft guidance explaining how combination product developers can demonstrate that their emergency-use injectors will reliably deliver drugs as intended in a life-threatening emergency.   The guidance specifically applies to emergency-use injectors that are prefilled or co-packaged with emergency drugs or biologics, such as those to treat anaphylaxis, and is intended to expand on FDA’s 2013 guidance Technical ...
  • Regulatory NewsRegulatory News

    Novartis Admits to ‘Mistake’ After Partial Clinical Hold Placed on Zolgensma Trial

    Novartis on Wednesday said that the US Food and Drug Administration (FDA) placed a partial hold on intrathecal clinical trials of its gene therapy Zolgensma (onasemnogene abeparvovec-xioi) for spinal muscular atrophy (SMA) patients based on findings in a small preclinical animal study.   On Thursday, a Novartis spokesperson told Focus that a draft report of the preclinical safety findings was presented to the AveXis safety management team last March and the company “...
  • RoundupsRoundups

    Asia Regulatory Roundup: Hong Kong Proposes Regulatory Framework for Cell and Gene Therapies

    Welcome to our Asia Regulatory Roundup, our weekly overview of the top regulatory news in Asia.   Hong Kong Proposes Regulatory Framework for Cell and Gene Therapies   Hong Kong has proposed legislation to create a regulatory framework for advanced therapy products (ATPs), such as interventions based on cells, genes and tissues. The plan is to amend existing laws to create licensing, labeling and record-keeping requirements for ATPs.   Work to update the law ...