All PMDA articles
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JournalNavigating the regulatory landscape for regenerative medicines in Japan
This is the final article in a series of three that critically analyse the differences and similarities between the regulation of advanced therapy medicinal products (ATMPs), also known as regenerative medicines…[1],[2]
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CPD SupplementUnderstanding the basics of ATMP regulation
Advanced therapy medicinal products (ATMPs) are biological products which comprise a diverse group of medicinal products that are often complex and diverse in nature. As these products are regularly developed for diseases of high unmet need, the requirements for evidence generation to support marketing authorisation often require innovative development plans. Within this continuing professional development supplement we investigate the regulation of ATMPs in the EU and US [see also Regulatory Rapporteur October 2020 and February 2020 for articles by Harriet Edwards on ATMPs].
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JournalGene therapy: regional assessment comparison
Axicabtagene ciloleucel (tradename YESCARTA) is an autologous chimeric antigen receptor T-cell (CAR-T) therapy that targets CD19 and is approved for the treatment of aggressive, relapsed or refractory forms of B-cell non-Hodgkins lymphoma (NHL). The active substance is composed of a patient’s cells that have undergone ex vivo modification, which causes ...
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JournalGlobal regulatory frameworks for the development of gene therapy products
Gene therapies have the potential to revolutionise the global healthcare system. Some of these products may offer curative benefits to patients with devastating conditions. This continuing professional development (CPD) supplement provides an overview of the regulatory framework for the development of gene therapy products in key markets.