Sarepta Therapeutics (SRPT) Status Update summary

FDA Label Expansion and Clinical Evidence

• ELEVIDYS received expanded FDA approval for Duchenne patients aged 4 and older, regardless of ambulatory status, with traditional approval for ambulatory and accelerated approval for non-ambulatory patients; all age and ambulation restrictions for patients 4+ are removed.

• ELEVIDYS is contraindicated in patients with deletions in exon 8 and/or exon 9 of the DMD gene.

• Clinical trials, including EMBARK, demonstrated significant benefits in key functional tests and biomarkers, with over 400 patients dosed across a broad age and weight range.

• Biomarker improvements include reduced CK levels and favorable MRI muscle/fat fraction changes, with further data to be presented at scientific meetings.

• Preexisting anti-AAVrh74 antibodies may reduce therapy effectiveness; baseline antibody testing is recommended.

Safety Information and Precautions

• Infusion-related reactions, including hypersensitivity and anaphylaxis, may occur; close monitoring during and after infusion is required.

• Acute serious liver injury and elevated liver enzymes have been observed; liver function should be monitored before and after infusion.

• Immune-mediated myositis and myocarditis have been reported; patients should be monitored for muscle and cardiac symptoms.

• Most common adverse reactions include vomiting, nausea, liver injury, fever, and thrombocytopenia.

• Studies are ongoing to address the 14-15% of patients with pre-existing antibodies, using imlifidase and plasmapheresis, with data expected next year.

Commercial Readiness and Manufacturing Capacity

• No near-term supply or manufacturing constraints are anticipated; over 70 infusion sites are prepared, exceeding initial goals.

• 75% of sites have already dosed patients, and additional capacity can be added as needed; site education and readiness have been a focus since 2018.

• Transition to a suspension manufacturing process is targeted for late 2026, aiming for greater efficiency and lower cost of goods.

• Sarepta collaborates with Roche for global access, with Sarepta handling US commercialization and Roche responsible internationally.

• The company is financially strong, with no immediate need to raise additional capital and tracking toward cash flow positivity.