Mutation

Sarepta’s Unusual Journey

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Before approving an investigational drug, safety and effectivity needs to be proven. Something different happened with Sarepta’s Muscular dystrophy drug Eteplirsen, as its luck changed completely when the U.S. Food and Drug Administration on 19th September 2016 approved Sarepta Therapeutics’ Exondys 51 (eteplirsen) injection to treat patients with Duchenne muscular dystrophy (DMD). Exondys51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD. This is the first drug to get approved for this rare disease.

Earlier in March, 2016, FDA had declared that it was not able to decide on Sarepta’s eteplirsen and needed more time to review. In making this decision, FDA considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease for these children and the lack of available therapy. Under the accelerated approval provisions, FDA required Sarepta Therapeutics to conduct a clinical trial to confirm the drug’s clinical benefit. In a complete turn of events, FDA approved Exondys 51 (eteplirsen) for the treatment of Duchenne muscular dystrophy (DMD), leading the stock to more than double in price in just a few days.

Sarepta’s drug has many health insurance providers are on board with it, others like Anthem Inc are still questioning whether it will do its customers any good — given the drug’s accelerated approval. Additionally, on October 5, Sarepta Therapeutics and Summit Therapeutics partnered to advance the development of novel therapies for the treatment of Duchenne muscular dystrophy.

Insight by:
Sadaf Javed
Associate Analyst
DelveInsight Business Research