2024-04-08 16:11:05 ET
Summary
- Sarepta Therapeutics generated $1.14 billion in revenues in 2023 from its four approved therapies for Duchenne Muscular Dystrophy.
- The company's gene therapy drug Elevidys faced concerns after a failed confirmatory study, causing a sharp market sell-off.
- Sarepta plans to push for a full approval and label expansion for Elevidys, targeting a broader patient population.
- The FDA will rule on Elevidys full approval and label expansion this June, but is success already baked into the share price?
Investment Overview
Sarepta Therapeutics ( SRPT ) markets and sells four of the six therapies that have been approved by the Food and Drug Agency ("FDA") to treat the disease Duchenne Muscular Dystrophy ("DMD"), a rare genetic disease characterized by progressive muscle degeneration. As we can see below, Sarepta's drugs generated $1.14bn of revenues for the company in 2023, up from $843.8m in the prior year.
According to Sarepta's 2023 10K submission:
The FDA granted accelerated approval for EXONDYS 51, VYONDYS 53, AMONDYS 45 and ELEVIDYS, respectively, as therapeutic treatments for Duchenne in patients who have a confirmed mutation in the dystrophin gene that is amenable to exon 51, exon 53, exon 45 skipping, and ambulatory pediatric patients aged four through five years with Duchenne with a confirmed mutation in the Duchenne gene, respectively.
EXONDYS 51 has been approved for marketing in the U.S., Israel and Kuwait, AMONDYS 45 in the U.S. and Kuwait, and VYONDYS 53 and ELEVIDYS have been approved for marketing only in the U.S. Our commercial phosphorodiamidate morpholino oligomer ("PMO") products are also available in additional countries through our early access programs ("EAPs").
The accelerated approvals for EXONDYS 51, VYONDYS 53 and AMONDYS 45 granted by the FDA were based on an increase in the surrogate biomarker of dystrophin in skeletal muscles observed in some patients treated with these products.
The accelerated approval for ELEVIDYS granted by the FDA was based on an effect on the surrogate endpoint of expression of the protein produced by ELEVIDYS. These products are subject to ongoing FDA requirements governing labeling, packaging, storage, advertising, promotion and recordkeeping, and we are required to submit additional safety, efficacy and other post-marketing information to the FDA.
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For further details see:
Sarepta Therapeutics: Another 'Hold' Call Ahead Of Some Key Catalysts