The Food and Drug Administration has awarded Sarepta Therapeutics a platform technology designation for its rAAVrh74 viral vector, representing one of the first such designations issued since the FDA launched this streamlined review pathway in 2024. The designation could accelerate development timelines and reduce costs for Sarepta's pipeline of gene therapies targeting rare muscular dystrophies.
Streamlining Gene Therapy Development
The platform technology designation program was conceived in 2023 and implemented by the FDA in 2024 to incentivize companies to use consistent drugmaking platforms across multiple therapeutic programs. For genetic medicines targeting rare diseases, this approach addresses a significant development challenge where each new therapy typically requires the same lengthy and expensive regulatory journey, despite using similar underlying technologies.
"This is one of the first programs to receive platform technology designation and an important recognition by FDA of the reproducibility and adaptability of this technology across multiple therapeutic programs," said Louise Rodino-Klapac, chief scientific officer and head of research & development at Sarepta.
To qualify for the designation, companies must demonstrate that their technology platform is well-understood and reproducible, and can support development of multiple drugs without compromising quality, manufacturing, or safety. The technology must also be incorporated in an FDA-approved drug with preliminary evidence showing potential for broader application.
Leveraging Elevidys Success
Sarepta's rAAVrh74 vector serves as the delivery mechanism for Elevidys, the company's already approved Duchenne muscular dystrophy gene therapy. The platform designation now allows Sarepta to use evidence previously gathered from Elevidys studies in future regulatory applications for other gene therapies using the same vector.
The designation specifically applies to SRP-9003 (bidridistrogene xeboparvovec), an investigational gene therapy for limb-girdle muscular dystrophy type 2E/R4. SRP-9003 is designed for systemic delivery to skeletal, diaphragm, and cardiac muscle, delivering a full-length beta-sarcoglycan transgene using the MHCK7 promoter chosen for robust cardiac expression.
According to Sarepta, SRP-9003 could be submitted to regulators later this year, with the platform designation potentially expediting the review process. The vector is also incorporated in at least two other programs targeting limb-girdle muscular dystrophy.
Investment and Development Implications
William Blair analyst Sami Corwin noted that the platform designation could accelerate development of Sarepta's follow-on gene therapy programs while lowering early research and development costs. The designation may also address investor concerns about Elevidys following a patient death associated with the therapy, as some had speculated about potential approval rescission.
However, the breadth of the designation's applicability remains uncertain. Sarepta management has indicated that manufacturing processes differ between Elevidys and SRP-9003 compared to other pipeline programs SRP-9004 and SRP-9005, which could limit the platform's utility across the entire portfolio.
Addressing Rare Disease Challenges
The platform technology approach is particularly relevant for genetic medicines, where many gene therapies target small patient populations with rare diseases. Developers often face situations where the same underlying technology could treat additional patient groups or similar diseases, but current regulatory pathways require starting the development process from scratch.
The designation enables sponsors to leverage prior platform data to support investigational new drug applications, new drug applications, or biologic license applications, potentially reducing development timelines and costs for subsequent programs using the established technology platform.
