FDA Grants Sarepta Therapeutics New Platform Technology Designation for Gene Therapy

The U.S. Food and Drug Administration (FDA) has awarded Sarepta Therapeutics a novel "platform technology designation" for its gene therapy delivery tool, potentially expediting future regulatory reviews for the company's genetic medicine pipeline. This development marks a significant milestone in the FDA's efforts to streamline the evaluation process for certain gene therapies.

Streamlining Gene Therapy Development

The platform technology designation, conceived in 2023 and implemented the following year, aims to incentivize pharmaceutical companies to utilize consistent drugmaking platforms across multiple programs. For Sarepta, this designation applies to its viral vector rAAVrh74, a crucial component in several of the company's gene therapy programs, including the FDA-approved Duchenne muscular dystrophy treatment, Elevidys.

Under this new designation, Sarepta may leverage previously gathered evidence from studies involving the rAAVrh74 vector in future regulatory applications. This approach could significantly reduce development timelines and early R&D costs for the company's gene therapy portfolio.

Implications for Rare Disease Treatment

The FDA's initiative is particularly relevant for genetic medicines targeting rare diseases. Many gene therapies are tailored to small patient populations, making the traditional drug development process both lengthy and expensive. The platform technology designation addresses this challenge by allowing companies to adapt their approved technology for treating additional patient groups or similar diseases without undertaking the full, conventional drug development journey.

To qualify for the designation, developers must first secure approval for a drug incorporating the technology and demonstrate its potential to accelerate the development of other medicines without compromising quality or safety. Once obtained, the designation is expected to yield "efficiencies in drug development, manufacturing, and review processes," according to the FDA's draft guidance.

Sarepta's Gene Therapy Pipeline

Sarepta's rAAVrh74 vector is not only used in Elevidys but also in SRP-9003, a limb-girdle muscular dystrophy treatment that may be submitted for regulatory review later this year. Additionally, at least two other muscular dystrophy programs in Sarepta's pipeline incorporate this vector.

However, the extent to which Sarepta can utilize this designation across its entire portfolio remains unclear. Management has indicated that Elevidys and SRP-9003 are produced differently from SRP-9004 and SRP-9005, two other limb-girdle gene therapies in development. This distinction may limit the applicability of the platform technology designation to certain programs within Sarepta's pipeline.