- Sarepta Therapeutics (SRPT, Financial) receives FDA clearance for the SRP-9005-101 (COMPASS) study for LGMD2C/R5, adding a fourth limb-girdle muscular dystrophy program to clinical trials.
- Enrollment and dosing have been completed in the SRP-9004-102 (DISCOVERY) study for LGMD2D/R3.
- The SRP-9003 (EMERGENE) phase 3 trial for LGMD2E/R4 is on track, with data expected by mid-2025, and potential accelerated approval confirmed by the FDA.
Sarepta Therapeutics, Inc. (SRPT) has announced pivotal advancements in its suite of clinical programs directed at different subtypes of limb-girdle muscular dystrophy (LGMD), marking a significant milestone in the company's gene therapy pipeline. The U.S. Food and Drug Administration (FDA) has granted clearance for the commencement of dosing in the SRP-9005-101 study for LGMD2C/R5, also known as gamma-sarcoglycanopathy.
Additionally, the biotech firm has finalized the enrollment and dosing processes in the SRP-9004-102 study for LGMD2D/R3, validating the safety and expression of the alpha-sarcoglycan protein. This study is crucial in proving the concept of Sarepta's investigational gene therapy approach targeting alpha-sarcoglycanopathy.
Furthermore, Sarepta's phase 3 trial involving SRP-9003 (EMERGENE) for LGMD2E/R4 has completed its enrollment and dosing phase. This trial is anticipated to deliver data by mid-2025. Notably, the FDA has affirmed eligibility for an accelerated approval pathway for this program, paving the way for a possible Biologics License Application (BLA) submission in the latter half of 2025.
With no currently approved disease-modifying treatments for any LGMD subtype, Sarepta's progression in their pipeline—covering over 70% of known LGMD cases—represents a beacon of hope for patients. The company's gene therapy programs span multiple LGMD subtypes, potentially securing Sarepta a pioneering position in these niche markets with substantial unmet medical needs.
