Sarepta's Elevidys Faces Setbacks Amid Safety Concerns and Leadership Changes in Biopharma Industry

Sarepta Therapeutics, a prominent player in genetic medicine, is grappling with challenges surrounding its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys. The company's recent earnings call revealed a complex landscape of patient hesitation, regulatory scrutiny, and industry-wide transformation.

Elevidys Sales Decline Amid Safety Concerns

Sarepta's CEO, Doug Ingram, addressed the company's first-quarter earnings, acknowledging a challenging period for both Sarepta and the broader biopharma industry. Following reports of a patient death after Elevidys treatment, some families have paused their treatment plans, seeking additional information. This hesitation, combined with a severe flu season and administrative delays at treatment sites, resulted in Elevidys missing sales expectations. The drug generated $375 million in sales during Q1, representing a 2% sequential decline and falling 11% below analysts' consensus estimates.

The company's Chief Customer Officer, Dallan Murray, warned that Elevidys sales in Q2 could be up to 20% lower than in Q1. Consequently, Sarepta has revised its 2025 total revenue projection to between $2.3 billion and $2.6 billion, down from the previous guidance of $2.9 billion to $3.1 billion.

Safety Profile and Regulatory Response

In March, Sarepta disclosed that a 16-year-old patient had died from acute liver failure following Elevidys treatment and a recent cytomegalovirus infection. This marks the first death among over 800 patients who have received the therapy in clinical trials or commercial settings. Sarepta has submitted a request to the FDA to include this case in Elevidys' label, with the agency planning to review the situation by year-end.

Louise Rodino-Klapac, Sarepta's R&D chief, stated that data so far suggest no correlation between adverse events and patient age, weight, or total Elevidys dose administered. Despite the setback, Ingram maintains that Elevidys still boasts "one of the most impressive safety profiles" among AAV-based gene therapies.

Industry Leadership Changes and Market Implications

The appointment of Vinay Prasad, M.D., a known biopharma industry skeptic, as the new leader for the FDA's Center for Biologics Evaluation and Research (CBER) has raised concerns among investors. Some analysts have even posited a worst-case scenario where Elevidys could be pulled from the market. Ingram, however, expressed confidence in the FDA's commitment to science-based decision-making and its mission to bring safe and efficacious therapies to patients.

Despite these challenges, Sarepta remains optimistic about Elevidys' long-term prospects. The company is focusing on educating the wider treating and referring physician landscape about the therapy's data, a process Murray acknowledges will take time. Ingram emphasized that top experts in the DMD field have not changed their treatment approach, and many families still view Elevidys' benefit-risk profile positively.