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  • Regulatory NewsRegulatory News

    EC Publishes New Guidelines on Good Clinical Practices for ATMPs

    Thanks to a 2007 regulation on advanced therapy medicinal products (ATMPs), the European Commission (EC) on Wednesday released new guidelines on the good clinical practice (GCP) requirements for these complex and innovative products that can include gene and cell therapies. The 15-page guidelines discuss clinical trial design, non-clinical studies, quality of the investigational ATMPs, the safe conduct of the clinical trial, upstream interventions on subjects and admini...
  • Regulatory NewsRegulatory News

    PIC/S Opens Consultations on Manufacturing ATMPs, Biological Medicinal Substances

    The Pharmaceutical Inspection Co-operation Scheme (PIC/S) is seeking comments on revisions to two parts of its GMP Guide (Annex 2A and Annex 2B) that deal with the manufacture of advanced therapy medicinal products (ATMPs) and biological medicinal substances and products for human use. PIC/S explains that draft Annex 2A accounts for international developments in the regulation of ATMPs, with particular attention to the European Commission guideline on GMP for ATMPs, whi...
  • Regulatory NewsRegulatory News

    EMA Offers Q&A on Exemptions From Batch Re-Testing for Imported ATMPs

    The question and answer (Q&A) document released by the European Medicines Agency (EMA) on Wednesday describes when an exemption from EU batch re-testing can be granted for imported advanced therapy medicinal products (ATMPs). The three-page Q&A begins by noting that a Qualified Person (QP) has to ensure each batch of imported ATMP is re-tested upon importation. But the QP can certify that the imported batch relies on controls conducted in a third country if the product ...
  • Regulatory NewsRegulatory News

    EMA Opens Consultation on Guideline for Advanced Therapies in Clinical Trials

    The European Medicines Agency (EMA) on Thursday opened for consultation a new guideline on the structure and data requirements for a clinical trial application for exploratory and confirmatory trials with advanced therapy investigational medicinal products (ATIMPs). The 53-page guideline addresses development, manufacturing and quality control, featuring sections on quality documentation (including parts on the active substance and the investigational medicinal product)...
  • Regulatory NewsRegulatory News

    Pharma Inspection Co-operation Scheme Criticizes Plan to Lower GMP Requirements for ATMPs

    The regulatory group known as the Pharmaceutical Inspection Co-operation Scheme (PIC/S) said in a letter released this week that it is “unanimously concerned” about the public health and safety impact of a new European Commission guideline that would lower good manufacturing practice (GMP) requirements for advanced therapy medicinal products (ATMPs). "By lowering the GMP requirements for ATMPs, the European Commission is not only exposing patients to an increased risk t...
  • Feature ArticlesFeature Articles

    Advanced Therapy Medicinal Products: An Evolving Regulatory Landscape

    This article was developed from a presentation at RAPS Regulatory Convergence in September 2016 in San Jose, California, US. It was the opening presentation in the "Global Developments in Cell and Gene Therapy" session and covered introductory Advanced Therapy Medicinal Product (ATMP) concepts from definitions to strategic considerations and challenges currently faced by developers in this field. Introduction Broadly speaking, Advanced Therapy Medicinal Products (ATM...
  • European Regulatory Roundup: UK Fines Pfizer for 2,600% Price Hike (8 December 2016)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. EC Appeases Some SMEs With Revised Advanced Therapy GMPs but Objections Remain The European Commission has received some favorable feedback on its draft good manufacturing practice (GMPs) guidelines for advanced therapy medicinal products (ATMPs). However, while small and medium-sized enterprises (SMEs) were broadly supportive of changes proposed in the draft, the commission...
  • Regulatory NewsRegulatory News

    European Commission to Overhaul Concept of ‘Similar’ Medicines Within Context of Orphan Legislation

    The European Commission on Friday proposed to review the concept of “similar medicinal products” in the context of its orphan legislation as part of a wider effort to adapt the text to technical progress. Fifteen years after the implementation of the orphan legislation, the commission says it is currently launching initiatives to improve the implementation of the regulatory framework with a view to ensuring timely access to medicinal products.  In this context, the Comm...
  • 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

    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...
  • 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 Releases Paper on Advanced Therapies to Assist Sponsors

    The European Medicines Agency (EMA) has released a new reflection paper on the classification of advanced-therapy medical products (ATMPs)-therapies made from genes and cells-which aims to clarify the legal basis for the classification of advanced therapies and provide information to applicants on how to submit applications for approval to EMA. In particular, the paper "provides information on how these medicines are classified as gene therapy, somatic-cell therapy, tiss...