There has been a surge in clinical development of Advanced Therapy Medicinal Products (ATMPs), also referred to as Cellular & Gene Therapy Products (CGTPs), both in the EU and the USA.
Many start-up companies, as well as large biopharmaceutical companies, are planning or have already entered manufacturing these cellular therapies or gene therapies (e.g., genetically engineered viral vectors, mRNA-lipid nanoparticles). While off to a slow start in market approval, the FDA has projected that by 2025 there could be up to 10-12 new ATMPs approved for the market each year.
While many CMC teams are familiar with the CMC regulatory compliance strategy for other biological products (e.g., monoclonal antibodies), they may not understand the significant differences and extra challenges in the CMC regulatory compliance strategy for ATMPs. These extra CMC challenges are due to the increased size and complexity of the nucleic acid-based biologics; the increased challenge in manufacturing of living biologic medicines (substantially manipulated patient cells, genetically engineered viruses and cells); and the limited size of the manufacturing runs (frequently patient-specific batches). This course will provide insights and practical guidance for the CMC teams to develop a cost-effective, risk-based CMC regulatory compliant strategy for the manufacture and control of ATMPs from first-in-human (FIH) clinical studies through market approval. The course emphasis will include the limited FDA and EMA CMC regulatory guidance. Real-world case examples of CMC regulatory compliance problems and delays affecting ATMPs will be discussed.