• 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...
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    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...
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    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-...
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    EU's First Stem Cell Treatment Recommended by EMA

    A major milestone in stem cell therapy was reached in December 2014 when a European Medicines Agency (EMA) committee recommended the conditional approval of Holoclar, the first stem-cell product to be recommended for approval in the EU. Background Holocloar, developed by Chiesi Farmaceutici S.p.A. and Holostem Terapie Avanzate, is an advanced-therapy medicinal product (ATMP) granted orphan designation for limbal stem cell deficiency (LSCD) caused by chemical or physic...
  • EMA Shines a Light into its Black Boxes of Regulatory: Its Review Committee Meetings

    US regulatory professionals have long benefited from meeting transcripts and other open meeting requirements that have meant that the inner workings and decisions of the US Food and Drug Administration's (FDA) various advisory committees are available for debate and dissection. Background This can be extremely useful for a range of stakeholders. Regulatory professionals seeking approval for similar products can tailor their applications to address the needs of committee...
  • Centralized Procedure to see Simplified Submission Process, Says EMA

    The European Medicines Agency (EMA) has announced that it has simplified the process by which marketing authorization applications (MAAs) are submitted to the agency, reducing the number of applications that marketing authorization holders (MAHs) need to submit during the centralized procedure. The changes comes just as EU regulators are in the midst of implementing the pharmacovigilance legislation, an EU-wide set of requirements intended to make it easier to track and ...
  • EMA Wants to Clarify Process for Developing Gene- and Cellular-Based Products

    The European Medicines Agency (EMA) is touting the ongoing efforts of its Committee for Advanced Therapies (CAT), a panel of expert advisors that EMA said expects to review between three and four marketing authorization applications (MAAs) in 2013. Of particular interest to the committee in the coming year: clarifying for small businesses the process by which they can get scientific advice to support regulatory approvals for advanced therapies derived from gene- and cellu...
  • EMA's Committee for Advanced Therapies to Assume More Responsibility Under Restructuring

    Officials at the European Medicines Agency (EMA) have announced the reorganization of two working groups within its Committee for Advanced Therapies (CAT), an advisory committee charged with evaluating advanced-therapy medicinal products (ATMPs). Under the new changes, both the Cell-based Products Working Party (CPWP) and the Gene Therapy Working Party (GTWP) will be transformed into ad hoc drafting groups, said EMA. In a statement, EMA explained the move is inte...
  • Paper: Small Companies Need Regulatory Assistance from EMA

    The European Medicines Agency (EMA) needs to assist companies-particularly small businesses, academic institutions and charities-in the development of advanced-therapy medicines, claims a new paper written by members of the Committee for Advanced Therapies (CAT) in the journal Molecular Therapies . These smaller drug development entities often lack the resources necessary to "navigate regulatory procedures," including clinical trials and application submissions. The pa...