Core Insights - Sarepta Therapeutics (SRPT) is experiencing a decline in share price following the announcement of a voluntary and temporary halt to all U.S. shipments of its gene therapy Elevidys, which is designed for Duchenne muscular dystrophy (DMD) [1][3] Company Developments - The pause in shipments is a proactive measure to address information requests and complete the safety labeling supplement process with the FDA, indicating the company's commitment to a science-based review [2][8] - Elevidys has been a significant revenue driver for Sarepta, contributing over half of the company's total revenues in Q1 2025, and the halt is expected to have a substantial negative impact on financial performance in the latter half of the year [3][8] - The decision to halt shipments follows the death of a patient in a clinical trial, marking the third patient death linked to Sarepta's gene therapies, which raises serious safety concerns [4][5][6] Regulatory Actions - The FDA has placed all of Sarepta's clinical studies for limb-girdle muscular dystrophy (LGMD) on hold due to the patient deaths associated with acute liver failure (ALF) [6][7] - The agency requested the voluntary stop of Elevidys shipments, which Sarepta initially resisted but ultimately complied with [7][8] Financial Restructuring - In response to the challenges, Sarepta announced a major restructuring plan that includes cutting 36% of its workforce, approximately 500 employees, to achieve annual savings of $120 million, alongside a reprioritization of its pipeline to save an additional $300 million per year [12][14] - The company is now focusing on its siRNA pipeline, which was acquired through a recent deal with Arrowhead Pharmaceuticals, targeting various conditions and expecting initial clinical data by year-end [14] Market Performance - Year-to-date, Sarepta's shares have plummeted by 89%, contrasting sharply with the industry's modest decline of 2.2% [7]

Core Insights - Shares of Sarepta Therapeutics (SRPT) fell 35.9% following the death of a patient in a trial for its gene therapy SRP-9004, which is aimed at treating limb-girdle muscular dystrophy (LGMD) Type 2D [1][9] - This incident marks the third patient death linked to Sarepta's gene therapies, with previous deaths occurring in non-ambulatory patients treated with Elevidys for Duchenne muscular dystrophy (DMD) [2][9] - The FDA has placed all of Sarepta's clinical studies for LGMD on hold and requested a halt to Elevidys shipments, which the company has refused, citing safety data [5][6][9] Company Developments - Sarepta has suspended Elevidys dosing for non-ambulatory patients and is considering enhanced immunosuppression to improve safety [3] - The company has lowered its net product revenue guidance for full-year 2025 to $2.3-$2.6 billion from $2.9-$3.1 billion due to safety concerns affecting drug uptake [4] - Sarepta announced a restructuring plan to cut 36% of its workforce, aiming for annual savings of $400 million by 2026 [13][15] Regulatory Actions - The FDA has revoked Sarepta's AAVrh74 platform technology designation, citing insufficient evidence to support its safety for multiple drugs [10] - The agency continues to assess risks associated with gene therapies based on this platform, including severe liver complications [10] Product Information - Elevidys is the first and only one-time gene therapy for DMD in the U.S., approved for individuals aged four years and older [11] - The therapy has received full approval for ambulatory DMD patients but only accelerated approval for non-ambulatory patients [11] Financial Impact - Sarepta's sales from Elevidys accounted for over half of its revenue in Q1 2025, and the recent safety concerns are expected to severely impact sales in the latter half of the year [4] - The company anticipates more than $100 million in cost savings this year despite incurring one-time charges of up to $37 million [15]