Healthcare Business

Odds of Sarepta Drug Approval Rise Sharply

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Shares of drugmaker Sarepta Therapeutics Inc. (NASDAQ: SRPT) went through the roof Monday morning following an announcement that the company plans to have a New Drug Application (NDA) filed with the U.S. Food and Drug Administration (FDA) by the end of 2014 for its muscular dystrophy drug eteplirsen. The stock is up more than 50% on the news.

Sarepta’s decision is based on a guidance letter from the FDA proposing that Sarepta submit its NDA for accelerated approval. The agency said in its letter that “with additional data to support the efficacy and safety of eteplirsen for the treatment of [Duchenne muscular dystrophy], a [new drug application] should be fileable.”

The company plans to begin several new clinical studies this year as a result of the FDA’s letter. The company’s chief medical officer said:

We are excited to have guidance from the FDA that allows us to move quickly into additional clinical trials with eteplirsen to confirm our current understanding of eteplirsen’s safety profile, its effect on dystrophin production, and its impact on clinical outcomes in DMD patients. We are particularly pleased that the FDA shares our interest in accelerating the clinical development of [eteplirsen].

Over the weekend, Barron’s noted that the FDA was facing pressure from patient advocates, medical experts and members of the U.S. Congress to get moving on approval for this drug. Duchenne muscular dystrophy affects about 15,000 boys in the United States, and more die from it than any other genetic disease. Victims usually die by the age of 20 or 25, Barron’s noted.

Barron’s suggested that if the drug is accepted for accelerated approval, Sarepta’s share price could double. That has not happened yet, but the shares were up about 52% to $36.88, in a 52-week range of $12.12 to $55.61. About 5 million shares had been traded early in the morning, compared with a daily average of around 1.3 million shares traded.

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