Core Viewpoint - Sarepta Therapeutics' stock experienced a significant decline of over 20% following the report of a patient's death after receiving Elevidys, its gene therapy for Duchenne muscular dystrophy (DMD) [1][4]. Company Overview - The patient who died was reported to have suffered from acute liver failure, a known risk associated with Elevidys. This incident marks the first death linked to the therapy, although the patient also had a recent CMV infection that may have contributed to the liver damage [2][3]. - Sarepta has communicated the event to health authorities and plans to update the prescribing information for Elevidys. The company asserts that the overall benefit-risk profile of the therapy remains positive, with over 800 patients treated [3]. Financial Impact - The decline in Sarepta's stock is attributed to concerns that this incident may deter doctors from prescribing Elevidys, potentially slowing its market adoption. Elevidys accounted for nearly 60% of the company's total revenues in Q4 2024, with projected net product revenues for the year between 3.1 billion, two-thirds of which are expected from Elevidys sales [4]. - Year-to-date, Sarepta's shares have decreased by nearly 40%, contrasting with a 6% growth in the industry [5]. Product Portfolio - Sarepta's portfolio includes four approved therapies for DMD, with Elevidys being the first one-shot gene therapy for the condition in the U.S. Since its launch in June 2023, Elevidys has shown significant sales potential, generating approximately 200 million in the previous year [7]. - The company has a partnership with Roche for the development of Elevidys, which has exclusive rights to market the therapy outside the U.S. [8]. - Other therapies in Sarepta's portfolio include Exondys 51, Vyondys 53, and Amondys 45, which collectively have the potential to address nearly one-third of all DMD patients in the U.S. [9].
SRPT Nosedives 27% on Patient Death Following DMD Therapy Infusion