A Look at Sarepta’s Exondys 51 Commercialization Efforts



Exondys 51

By applying its proprietary platform technologies, Sarepta Therapeutics (SRPT) is able to target a range of diseases and disorders. The company’s first product on the US market, the Exondys 51 injection, received accelerated approval from the US FDA in September 2016. The injection is targeted for treating Duchenne muscular dystrophy (or DMD).

Sarepta’s exon-skipping product candidates include eteplirsen, golodirsen, casimersen, and SRP-5051. In addition, Sarepta is also working with different strategic partners under various agreements to research and develop multiple approaches to DMD. These partners include Nationwide Children’s Hospital, Genethon, Duke University, and Summit.

European approval

Sarepta Therapeutics submitted a marketing authorization application (or MAA) to the European Medicines Agency (or EMA) in November 2016 for eteplirsen. This application was validated in December 2016. Sarepta is working with the EMA in its review process and anticipates completion of the review and a final decision in 2018.

Focus on market reach

Sarepta Therapeutics has initiated a market access program (or MAP) for eteplirsen in key countries in Europe, North America, and South America as well as Asia where it has not yet received approval. This move provides a mechanism through which physicians can prescribe the injection within their professional responsibility for patients that meet the pre-specified medical and other criteria and can secure funding.

Sarepta shipments through this program have started, and the program continues to expand to include more countries. Additionally, it has contracted with third-party distributors and service providers for the distribution of the injection in areas outside the US like Israel and other countries in the Middle East.

In the next part of the series, we’ll take a look at Sarepta’s agreement with Myonexus Therapeutics.

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