Sarepta Therapeutics (SRPT, Financial) saw a 36% surge after the FDA approved an expanded indication for ELEVIDYS. The label now includes individuals with Duchenne muscular dystrophy (DMD) aged 4 and older.
- DMD is a rare genetic disease affecting mainly males, causing progressive muscle weakness and often leading to fatal respiratory or cardiac failure by the mid-20s.
- The FDA confirmed ELEVIDYS' benefits for ambulatory patients, granting traditional approval for those aged 4+. Additionally, the FDA granted accelerated approval for non-ambulatory patients, pending verification from the ENVISION trial.
- The expanded approval now covers both ambulatory and non-ambulatory children aged 4 and above, providing a treatment option for the majority of boys and young men with DMD.
- Sarepta highlighted the expansion's significance, stating it could benefit over 80% of diagnosed DMD patients, marking a major advancement in treatment options.
Investors are enthusiastic about the commercial potential of this expansion. Sarepta is prepared for a robust launch without supply constraints, despite the drug's high cost. This news follows Pfizer's (PFE, Financial) recent DMD study setback, solidifying Sarepta's dominant position in the DMD market.
