Core Insights - The FDA has granted platform-technology designation to Sarepta Therapeutics' rAAVrh74 viral vector, which is used in the investigational gene therapy SRP-9003 for limb-girdle muscular dystrophy (LGMD) [1][7] - This designation allows Sarepta to reuse clinical and manufacturing data across multiple therapies utilizing the same viral vector, potentially expediting development timelines [2][3] Company Developments - SRP-9003 is currently in a phase III EMERGENE study targeting LGMD type 2E/R4, with primary endpoints focused on beta-sarcoglycan protein expression [4] - Positive data from the ongoing study could lead to a regulatory filing for accelerated approval by the end of the year [5] - Despite the potential for SRP-9003, Sarepta's stock has seen a significant decline of 68% year-to-date, contrasting with a 4% decline in the industry [6] Recent Challenges - Sarepta faces negative sentiment due to safety concerns surrounding its gene therapy Elevidys, which is the first one-time treatment for Duchenne muscular dystrophy (DMD) [9] - A recent incident involving a patient's death post-treatment has led to a clinical hold on Elevidys studies by the EMA, raising apprehensions about market adoption [10][12] - Elevidys has generated approximately $821 million in sales for 2024, a substantial increase from $200 million the previous year, but safety concerns have prompted a revision of 2025 sales guidance to $2.3–2.6 billion from an earlier forecast of $2.9–3.1 billion [11][13] Strategic Partnerships - Elevidys was developed in collaboration with Roche, which holds exclusive rights to market the therapy outside the U.S. [14]
FDA Grants Platform Technology Tag to Sarepta's Gene Therapy Vector