Sarepta Therapeutics, a leader in precision genetic medicine for rare diseases, has announced its financial results for the second quarter of 2024.
“This quarter marks a groundbreaking milestone in Duchenne muscular dystrophy (DMD) treatment, comparable to the discovery of dystrophin as the disease’s cause in 1986. Thanks to compelling clinical evidence, the FDA has expanded access to our gene therapy, ELEVIDYS, for patients aged 4 and older, granting traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients. We are excited to present comprehensive data on ELEVIDYS at the 29th Annual Congress of the World Muscle Society in October, showcasing improvements in muscle health through muscle and cardiac MRI data and other biomarkers,” said Doug Ingram, President and CEO of Sarepta Therapeutics. “We are unmatched in our capability to launch Duchenne therapies and support the community through education, access, and reimbursement. Our preparations for the broadened launch have exceeded even our most optimistic expectations. For Q2, net product revenues from our four approved therapies reached $360.5 million, a 51% increase from the same period last year. Our PMO products—EXONDYS 51, VYONDYS 53, and AMONDYS 45—generated $238.8 million, while ELEVIDYS contributed $121.7 million. We anticipate significant growth in the latter half of 2024 as families complete the enrollment-to-infusion process. Looking ahead, based on our early launch signals and ongoing PMO performance, we project net product revenues for 2025 will range between $2.9 and $3.1 billion.”
Second Quarter 2024 and Recent Developments:
- FDA Expands ELEVIDYS Indication: On June 20, 2024, the FDA broadened ELEVIDYS’s approval to include DMD patients aged 4 and older with a confirmed DMD gene mutation. Ambulatory patients received traditional approval, while non-ambulatory patients were granted accelerated approval, pending confirmation of clinical benefit in a follow-up trial. ENVISION (Study SRP-9001-303), a Phase 3 study, is underway to verify ELEVIDYS’s efficacy in non-ambulatory patients.
- European Review of ELEVIDYS: On June 24, 2024, Roche announced the EMA has begun reviewing the ELEVIDYS marketing authorization application for ambulatory patients aged 3 to 7 years, with expected approval in 2025. Roche will handle ELEVIDYS commercialization outside the U.S.
- Upcoming Data Presentation: In October, Sarepta will present data from its SRP-9001 program at the 29th Annual Congress of the World Muscle Society in Prague. The agenda is available at WMS 2024.
- Long-Term ELEVIDYS Studies: Sarepta is executing long-term follow-up studies for ELEVIDYS, including ENDURE (Phase 4) and EXPEDITION (Phase 3), to track safety and efficacy over extended periods.
- FDA Fast Track Designation for SRP-9003: The FDA has granted Fast Track designation to SRP-9003, a gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E/R4). This designation aims to expedite development and review of therapies for serious conditions with unmet needs.
- Dr. Jerry R. Mendell Recognized by TIME: Dr. Jerry R. Mendell, a leading neuromuscular researcher and gene therapy pioneer, was named to TIME’s inaugural 2024 TIME100 Health list for his significant contributions to genetic medicine and treatment of neuromuscular diseases, including Duchenne muscular dystrophy.
