Officials from the US Food and Drug Administration’s (FDA) Office of Therapeutic Products (OTP) within the Center for Biologics Evaluation and Research (CBER) urged developers of gene therapies to avoid implementing manufacturing process changes during later pivotal studies to avoid problems later, such as products being out-of-specification (OOS).
So asserted Graeme Price, a team lead for the Gene Therapy Branch 1 within OTP, who spoke at a virtual town hall on 25 April to answer stakeholder questions related to chemistry, manufacturing and controls (CMC) for gene therapy products as part of investigational new drug applications (INDs) and licensure applications.
The meeting also addressed differences between characterization and release testing, when assays need to be qualified, empty capsids and FDA’s expectations for potency testing.
OTP was formerly called the Office of Tissues and Advanced Therapies (OTAT) and was renamed last September and reorganized to a “Super Office” to meet its growing workload and new commitments under the Prescription Drug User Fee Act (PDUFA VII) agreement. (RELATED: FDA elevates OTAT to “Super Office” within CBER, Regulatory Focus 30 September 2022)…
