AAV Vector Consortia Address Quality Assays, Endpoints

While drug regulators are “conservative” by nature, the European Medicines Agency wants to help companies rather than rip their dossiers “into little pieces,” a senior regulatory assessor says.

The clarity on quality topics provided by the CMC Readiness Pilot (CDRP) is worth the work of preparing a comprehensive development review and multiple meetings, participants from Intellia, Bicycle Therapeutics and Bristol Myers Squibb said during a Duke Margolis meeting.

The FDA is talking with cell and gene therapy staff about the importance of review timeline alignment for products accepted into the Commissioner’s National Priority Voucher program, CBER’s Katherine Szarama said.

Sponsors may need to pursue more rapid CMC development to accommodate the faster pace of the clinical program, the FDA said in a new expedited pathways guidance, which also addresses product changes after RMAT designation and the use of real-world evidence to confirm clinical benefit.