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Sarepta Awaiting Approval for Exondys 51 in Europe

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European market approval

On December 20, 2016, Sarepta Therapeutics (SRPT) announced that the MAA (marketing authorization application) for Exondys 51 (eteplirsen) for Duchenne muscular dystrophy (or DMD) patients above four years of age with mutations amenable to exon-51 skipping was accepted by the EC (European Commission). After receiving initial feedback from the EC related to the application, the company requested a six-month clock stop to the review to collect additional data. Since the review clock has been restarted, Sarepta anticipates the European Medicines Agency’s decision in the first half of 2018.

To allow patients in international markets access to investigational therapies before regulatory approval,  Sarepta initiated a managed access program in July 2017. Also known as an early or expanded access program, it will allow physicians in Europe, North America, and South America to prescribe Exondys 51 based on their informed assessment. Currently, this program is operational in 28 countries across the world. Sarepta expects to start earning revenue from this program in late 4Q17. Sarepta accounts for 0.05% of the iShares Russell 2000 Value ETF’s (IWN) portfolio.

European commercialization efforts

Sarepta is involved in creating infrastructure for the commercial launch of Exondys 51 in Europe. The company has already hired a head of operations in the EU (European Union) and has opened its EU headquarters in Zug, Switzerland. The company is also working on scaling its manufacturing capabilities to meet the potential demand in future years. To create awareness for the drug, Sarepta has conducted detailed market research and has identified the key opinion leaders in the market. The company has also settled multiple major intellectual property and legal initiatives in the market.

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