SRPT Stock Down After EMA Places Clinical Hold on DMD Gene Therapy

Core Viewpoint - Sarepta Therapeutics' shares fell over 6% following the European Medicines Agency (EMA) placing a clinical hold on studies for Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD), due to a patient's death post-treatment [1][2]. Company Overview - Elevidys is a one-shot gene therapy developed in collaboration with Roche, and it is the first of its kind for DMD in the United States [4][9]. - The therapy has shown significant sales potential, contributing nearly 60% to Sarepta's total revenues in Q4 2024, with expectations of $2.9-$3.1 billion in net product revenues for 2025, two-thirds of which are anticipated from Elevidys [6]. Clinical Developments - The clinical hold was initiated after a patient's death attributed to acute liver failure, with ongoing investigations into the potential role of a recent CMV infection [2][3]. - Enrollment and dosing in three studies evaluating Elevidys at EU sites have been paused until the cause of death is fully analyzed [3]. Market Impact - Concerns regarding the clinical hold may lead to increased hesitance among doctors to prescribe Elevidys, potentially hindering its market adoption in the U.S. and delaying its launch in the EU [5]. - Year-to-date, Sarepta's shares have decreased nearly 52%, contrasting with a 1% growth in the industry [7]. Product Portfolio - Sarepta's portfolio includes four approved therapies for DMD, with Elevidys being a major contributor to revenue growth, having generated approximately $821 million in 2024 compared to $200 million the previous year [9][10]. - Other therapies in the portfolio, such as Exondys 51, Vyondys 53, and Amondys 45, have also seen strong demand and are expected to address nearly a third of all DMD patients in the U.S. [10].