PDA EU00190 CMC Regulatory Compliance Strategy for ATMPs – CGTPs

Chemistry, Manufacturing & Control (CMC) regulatory compliance strategy encompasses all of those activities that are required/expected to be carried out by a regulatory authority to protect the patient from the manufactured drug product. Such activities involve Genetic Development (gene development, cell clonality), Process Development (design of the manufacturing process), Analytical Development (design of the release/stability test methods), Manufacturing (carrying out the upstream and downstream, and drug product processes), Quality Control (testing to meet specifications), Quality Assurance (cGMP compliance) and Regulatory Affairs (communication with the authorities). Compared to other biologic drug products, the coordination and timely completion of all the activities by all these CMC groups has never been more needed than for ATMPs/CGTPs.

While many CMC teams are familiar with the CMC regulatory compliance strategy for other drug products (e.g., chemical drugs, monoclonal antibodies), they may not appreciate the significant differences and extra challenges in the CMC regulatory compliance strategy for ATMPs/CGTPs. These extra CMC challenges are due to the increased size and complexity of these biologics (infectious AAV viral vectors, mRNA non-viral vectors, living genetically modified patient cells), as well as the limitations of the manufacturing processes (e.g., limited number of batches, increased biological variability).

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/CGTPs from first-in-human (FIH) clinical studies through market approval. The course emphasis will include CMC regulatory guidance (FDA, EMA and ICH), as well as real-world case examples illustrating CMC regulatory compliance delays in advancing clinical development.