Sarepta Secures First FDA Platform Designation for Gene Therapy Delivery Technology

The U.S. FDA has granted Platform Technology Designation to Sarepta Therapeutics’ rAAVrh74 viral vector, the delivery component used in SRP-9003, an investigational gene therapy for limb-girdle muscular dystrophy type 2E/R4 (LGMD2E). This marks reportedly the first public confirmation of such a designation by a company, under a regulatory pathway designed to streamline development of therapies that share a common technological basis.

The designation is part of a new regulatory framework created under Section 506K of the Food, Drug, and Cosmetic Act and clarified in FDA’s May 2024 draft guidance. The Platform Technology Designation Program is intended to support efficiencies in drug development, manufacturing, and application review by recognizing technologies—such as vectors, sequences, or delivery methods—that are well-characterized and reproducibly deployed across multiple products.

To qualify, a platform must demonstrate reliability across programs without compromising safety or product quality. Sarepta’s rAAVrh74 has been used in multiple clinical-stage gene therapies, including those for Duchenne muscular dystrophy, supporting its eligibility.

According to the FDA, it expects to grant approximately ten such designations annually. Sarepta is the first company to publicly confirm receiving this status. The designation enables more streamlined INDs, BLAs, and NDAs for future programs using the rAAVrh74 vector, signaling FDA recognition of the platform’s technical consistency.

This precedent is expected to encourage other gene therapy developers to pursue platform designations for reusable components and may shift how regulatory agencies support modular development across rare disease pipelines.

Topics: Novel Therapeutics