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...

The European Commission report released Wednesday dissects the similarities in how gene-, cell- and tissue-based advanced therapies are regulated across the four different geographic regions, though ongoing research projects in such therapies are heavily concentrated in the US and Japan. The 327-page report highlights the “high degree of convergence” in the regulation of advanced therapies across the US, Canada, South Korea and Japan, noting that only Japan has enacted (...

The European Medicines Agency (EMA) on Friday released a report outlining the concerns and regulatory changes described by developers, manufacturers and investors in advanced therapy medicinal products (ATMPs), which include gene therapies, tissue-engineered products and somatic cell therapies, at a meeting last week. The report notes that recurring themes included the need for early interaction with regulators and guidance, more transparency, greater harmonization betwe...

The European Medicines Agency (EMA) on Tuesday released a host of classification decisions for advanced therapies being developed to treat various types of tissue damage, including burns, scars and non-healing wounds. The agency, through its Committee for Advanced Therapies (CAT), offers sponsors an optional consultation to determine whether a product meets the criteria for an advanced-therapy medicinal product (ATMP) . The classification procedure is laid out in Arti...

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...

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...

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...

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-...

Studying the history of regulatory approvals can provide insights into regulator expectations for new products. The authors undertook an investigation to examine the data sets on which previous US Food and Drug Administration (FDA) approvals for melanoma treatment products were based to determine whether trends have changed in the level of evidence provided. Melanoma was chosen due to its relevance to ongoing projects by the authors. Methodology The authors obtai...

Merck’s new skin cancer treatment has become the first product approved under a new accelerated review process by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA). The product, Keytruda, has been shown to be effective in slowing the progression of advanced melanoma, a deadly form of skin cancer with few treatment options. Accelerated Approval Keytruda is the first product to be approved under MHRA’s early access to medicines scheme (EAMS), whic...

UK melanoma patients still cannot get access to BMS' Ipilimumab (Yervoy), approved for Europe-wide marketing on 13 July 2011, or Roche's Vemurafenib (Zelboraf), approved on 20 February 2012.  The barrier to patient access has been the UK cost containment agency, the National Institute for Health and Clinical Excellence (NICE), which admits both new drugs are "breakthrough treatments" for melanoma patients.  But NICE has refused to clear the drugs for...

Draft guidance issued by the National Institute for Health and Clinical Excellence (NICE) on 15 June 2012 does not recommend the use of Roche's new melanoma drug, vemurafinib, by the National Health Service (NHS). "Vemurafenib is an expensive drug and its long term benefits are difficult to quantify," Sir Andrew Dillon, Chief Executive of NICE said. The data submitted by Roche, which compared vemurafenib to the current standard treatment using dacarbazine, showed...