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  • Regulatory NewsRegulatory News

    EMA Reflects on Advanced Therapy Classification

    The European Medicines Agency (EMA) has released a new reflection paper discussing how products are classified as advanced therapy medicinal products (ATMPs). Background In the EU, medicinal products derived from genes, cells or tissues are classified as ATMPs. Because these areas of biomedicine are still in early stages of development and are fundamentally different from other types of medicine, the European Commission (EC) regulates them differently. ATMPs are define...
  • Regulatory NewsRegulatory News

    AmpliPhi Biosciences Gets First cGMP Certification for Bacteriophage Facility

    AmpliPhi Biosciences announced Wednesday it has received the first ever good manufacturing practices certification for its bacteriophage facility in Slovenia. Background Bacteriophages are naturally occurring viruses that are capable of fighting bacteria. Phage therapy has been around for nearly a century, following the co-discovery of bacteriophages by Frederick Twort and Felix d'Hérelle in 1915 and 1917, respectively. Shortly thereafter, scientists began developing...
  • Regulatory NewsRegulatory News

    EMA Issues New Guideline on Gene Therapy

    The European Medicines Agency (EMA) is seeking feedback on a new draft guideline aimed at clarifying the scientific evidence necessary to support the authorization of new gene therapies. Background Gene therapy works by modifying a patient's genes to correct for genetic issues. Researchers have been studying gene therapy for the past 30 years, but few products have reached advanced stages of development. Because so few gene therapies have been submitted for authorizatio...
  • Regulatory NewsRegulatory News

    Following Negative Comments by Reviewer, EU Committee OK's Gene Therapy

    Last month, German regulators postponed their review of UniQure's gene therapy Glybera after the European Medicines Agency (EMA) rapporteur assigned to the product claimed new data showed Glybera lacked efficacy and called for it to be reassessed . Now, EMA's Committee for Advanced Therapies (CAT) has completed its assessment, and is maintaining its positive opinion of Glybera, Reuters reports. Background Glybera is the first gene therapy approved for use in the E...
  • Regulatory NewsRegulatory News

    Senators Seek to Expedite FDA Review of 'Breakthrough' Medical Devices

    Medical device manufacturers may soon benefit from an expedited review pathway now only afforded to "breakthrough" pharmaceutical products if a bipartisan trio of US legislators have their way. Background On 24 April 2015, Sens. Richard Burr (R-NC), Michael Bennet (D-CO) and Orrin Hatch (R-UT) introduced the Advancing Breakthrough Devices for Patients Act ( ABDP Act ) . As written, the law is heavily based on the "breakthrough therapies" section ( Section 902 ) of th...
  • Regulatory NewsRegulatory News

    First EU-Authorized Gene Therapy Hits Snag, Benefit Assessment Delayed in Germany as EMA Reviews New Report

    Regulators at the German Federal Joint Commission (G-BA) have postponed their review of the gene therapy Glybera, Reuters reports . The decision was made after the European Medicines Agency (EMA) rapporteur assigned to Glybera found the product lacked efficacy and called for it to be reassessed. Glybera, which treats a rare condition called lipoprotein lipase deficiency (LPLD), made headlines both for being the first gene therapy approved in Europe and for its record-...
  • Feature ArticlesFeature Articles

    Leveraging Expedited Programs for Drugs and Biologics for Serious Conditions and Rare Diseases

    In recent years, the US Food and Drug Administration (FDA) has made great advances in the review of new drugs. The US now is reported to lead the world in both timeliness and quantity of noteworthy new drug approvals. 1 This trend is due in part to FDA’s ongoing commitment to improve the drug development process and establish robust, efficient and predictable development programs. As a result, products demonstrating a positive benefit-risk profile and appropriate e...
  • Feature ArticlesFeature Articles

    FDA’s Expedited Programs for Serious Conditions: An Overview

    For some serious or life-threatening diseases, few therapeutic options exist. Once a promising therapy is discovered, establishing its clinical benefit and safety profile is necessary but can be time consuming. Standard drug development programs involve formulating and manufacturing the drug product, characterizing the new drug, gathering adequate evidence on its performance, evaluating the safety risks and confirming the effects observed in the early clinical trial...
  • Regulatory NewsRegulatory News

    FDA Wants Sponsors of Biotech Products to Assess Product's Impact on Environment

    The US Food and Drug Administration's (FDA) Center for Biologics Evaluation and Research (CBER) has released a new draft guidance document meant to clarify when companies need to conduct an Environmental Assessment (EA) for gene therapy and other recombinant products. Background Under 21 CFR 25 of the Code of Federal Regulations , most pharmaceutical and biotechnology companies are required to submit EAs as part of their approval package. The assessments are gene...
  • Cell and Gene Therapy Guidance Finalized by FDA, Calls for Early and Frequent Communication

    The US Food and Drug Administration (FDA) has announced the final release of a guidance intended to clarify the processes by which cellular and gene therapy (CGT) products should be assessed prior to human testing. Background CGTs are essentially used to make therapeutic changes to a person's cells, tissue or genes by injecting a patient with altered DNA, cells or entire organs. The products include cellular therapies, gene therapies, therapeutic vaccines, xenotransplan...
  • FDA Extends Comment Period on Cell and Gene Therapy Guidance by Six Months

    Manufacturers of cell therapy (CT) and gene therapy (GT) products will have six months of additional time to comment on a July 2013 draft guidance released by the US Food and Drug Administration (FDA), regulators announced today. Background In July 2013, FDA's Center for Biologics Evaluation and Research (CBER) released a draft guidance it said would help industry in designing early-stage clinical trials for CTs and GTs. The guidance was specifically aimed at investiga...
  • Safety Main Challenge to Preparing INDs for Cellular and Gene Therapy Products, Says FDA

    The US Food and Drug Administration (FDA) has released a new draft guidance document intended to outline the considerations industry should take into account when designing early-phase clinical trials for cellular therapy (CT) and gene therapy (GT) products. Background The draft guidance, released on 2 July 2013 by FDA's Center for Biologics Evaluation and Research (CBER), is specifically targeted at investigational new drug applications (INDs), which are used by compan...