Sarepta Therapeutics has achieved a significant milestone, receiving the U.S. FDA’s Platform Technology Designation for its rAAVrh74 viral vector. This designation, a first of its kind publicly documented for a gene therapy vector, is expected to accelerate the development and review of multiple gene therapies, particularly for rare diseases like Limb Girdle Muscular Dystrophy.
FDA Recognizes Sarepta’s Gene Therapy Platform
On June 4, 2025, Sarepta Therapeutics announced that the U.S. Food and Drug Administration (FDA) granted Platform Technology Designation to its rAAVrh74 viral vector. This vector is a key component in SRP-9003 (bidridistrogene xeboparvovec), an investigational gene therapy for Limb Girdle Muscular Dystrophy Type 2E/R4 (LGMD2E/R4). The designation signifies the FDA’s recognition of the technology’s reproducibility and adaptability across various therapeutic programs.
Key Takeaways
- Streamlined Development: The Platform Technology Designation aims to streamline drug development, manufacturing, and review processes for drug product applications by allowing sponsors to leverage prior data from the designated platform.
- Broad Applicability: To qualify, a technology must be well-understood, reproducible, and capable of supporting the development of multiple drugs or biologics without adverse effects on quality, manufacturing, or safety.
- Impact on Sarepta: For Sarepta, this designation is particularly favorable, as it is expected to accelerate the development of its follow-on gene therapy programs and potentially reduce early research and development expenses. The rAAVrh74 vector is also used in Sarepta’s already-approved Duchenne muscular dystrophy gene therapy, Elevidys.
- FDA’s Evolving Stance: This move aligns with recent statements from FDA leaders, including Commissioner Marty Makary, who have expressed support for regulatory flexibility and accelerated pathways for rare disease therapies, especially those with plausible mechanisms.
Accelerating Rare Disease Therapies
Louise Rodino-Klapac, Ph.D., Sarepta’s chief scientific officer, highlighted the importance of this designation, stating it underscores the consistency of data observed with AAVrh74 in multiple clinical programs. This recognition is a testament to Sarepta’s commitment to expediting potentially transformative treatments for patients with rare genetic diseases.
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Sources
- U.S. FDA Grants Platform Technology Designation to the Viral Vector Used in SRP-9003, Sarepta’s
Investigational Gene Therapy for the Treatment of Limb Girdle Muscular Dystrophy Type 2E/R4, Sarepta Therapeutics. - Sarepta Wins FDA’s Platform Technology Designation, Marking ‘Critical Shift’ in Gene Therapy Development –
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