Sarepta Therapeutics’ Elevidys Approval Expansion to Drive Revenue to $3.4B by 2028

Sarepta Therapeutics (NASDAQ: SRPT) has solidified its position as a leading player in treating Duchenne muscular dystrophy (DMD) following the label expansion for its gene therapy, Elevidys in June earlier this year. DMD is a progressive disorder that leads to muscle degeneration and weakness. Elevidys, a one-time gene therapy, aims to treat the root cause of DMD by delivering a functional, shortened version of dystrophin—a protein essential for maintaining muscle cell structure—into the muscle tissue. The absence of dystrophin is the primary cause of the symptoms seen in DMD.

Initially granted accelerated approval in June 2023 for ambulatory patients aged 4 to 5 years with a confirmed DMD gene mutation (those able to walk without mobility aids), Elevidys has since received traditional approval for ambulatory individuals aged 4 and older. Additionally, it now has accelerated approval for non-ambulatory patients in the same age group, all with confirmed DMD gene mutations.

This expanded approval is particularly important for Sarepta Therapeutics because it includes non-ambulatory patients, a group with significant unmet medical needs, and greatly increases the number of eligible patients for Elevidys. Elevidys is projected to generate $1.7 billion in revenue in 2024, up from $1.2 billion last year. Visible Alpha consensus shows analysts forecast the broader approval could lead to peak annual U.S. sales of $3.2 billion by 2028, with global peak sales projected at $3.4 billion.

Sarepta’s position in the DMD market has been further bolstered by setbacks for competitors. Pfizer’s (NYSE: PFE) Fordadistrogene Movaparvovec (FoMo) failed in Phase III trials, removing a significant near-term competitive threat to Elevidys. While other gene therapies for DMD, such as Regenxbio’s (NASDAQ: RGNX) RGX-202 and Solid Biosciences’ (NASDAQ: SLDB) SGT-003, as well as possible CRISPR-based treatments, are in development, they are still in the early stages and are unlikely to challenge Sarepta’s market dominance in the near future. This competitive landscape extends Sarepta’s first-mover advantage in the DMD gene therapy space. In 2024, Elevidys is estimated to account for 42% of the company’s overall revenue, with revenue share expected to increase to 76% by 2027. Roche (SIX: ROG) has exclusive rights to launch and commercialize Elevidys outside the U.S.