Sarepta’s ELEVIDYS Gaining Ground Amidst Anticipation for Expanded Label Approval

- **Thriving Launch**: Despite a limited FDA approval for treating ambulatory children aged 4 to 5 with Duchenne muscular dystrophy (DMD), Sarepta Therapeutics' ELEVIDYS has shown promising early commercial success.
- **Label Expansion Efforts**: Sarepta is actively pursuing a broader label for ELEVIDYS, submitting an efficacy supplement to expand usage beyond the initial age range and seeking priority review from the FDA.
- **Accelerated Approval Conversion**: Alongside the label expansion, Sarepta hopes to convert ELEVIDYS' accelerated approval to a traditional one, highlighting the importance of both goals.
- **Challenges**: The restricted patient population poses unique operational hurdles, such as identifying newly diagnosed cases among young children and managing the pre-treatment process, given the narrow window before children age out of eligibility.
- **Strong Market Performance**: ELEVIDYS generated $200.4 million in total sales during Q4 2023 alone, indicating a successful commercialization despite the restricted label.
- **Clinical Trial Support**: Results from the EMBARK phase 3 clinical trial in DMD patients aged 2 years and older contribute to the evidence supporting the label expansion.

Sarepta Therapeutics is optimistic about ELEVIDYS' performance while awaiting the FDA's decision regarding the expanded label, which they believe aligns with their mission to offer effective treatments for rare diseases like DMD.