FDA tentatively approves first drug for muscular dystrophy

Federal health regulators have granted tentative approval to a highly contested drug for muscular dystrophy that has become a flashpoint in the debate over patient access to experimental medicine.

Eteplirsen has had a controversial history with the FDA, and approval had been up in the air.

The U.S. Food and Drug Administration on Monday gave accelerated approval to the first drug for the crippling disease Duchenne muscular dystrophy, from Sarepta Therapeutics Inc., after sharp disagreements within the agency.

"The FDA has concluded that the data submitted by the applicant demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in some patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping", the statement said.

Shares in Sarepta skyrocketed by over 90% at one point Monday morning, as the FDA's decision resolved a long-standing question of whether the company would be sent back to the drawing board.

Several companies have pulled out of the space after the FDA made clear there was no path forward for approval for their drugs. "But it wasn't timely enough". DMD patients are typically wheelchair-bound by their early teens and often die in their 20s or 30s. With the help of its supporters, MDA plans to double its research spend targeting treatments and clinical trials by the year 2020.

The approval was based on a company study of just 12 boys.

In his review of the scientific dispute, Califf wrote that "Unger had an adequate opportunity to present his scientific concerns within the Center for Drug Evaluation and Research, and that Dr. Woodcock considered all relevant evidence in making her decision". A coalition of women's groups backed by the manufacturer, Sprout Pharmaceuticals, packed an advisory committee meeting with women who testified to their desperate need for the pill. The FDA missed its initial deadline to decide on whether to approve the drug and requested more data from the company, fueling a rollercoaster of rumors about whether the drug would ultimately be approved.

"In rare diseases, new drug development is especially challenging, due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders", Woodcock said. The drug, now known as Exondys 51 (eteplirsen), is the first approved treatment for DMD. "In particular, such action eviscerates the FDA's longstanding requirement that there be substantial evidence of effectiveness for new drugs-even drugs for serious rare diseases-before they are marketed". The 52 week high shares of Sarepta Therapeutics have reached is 56.18 whilst the 52 week low for the company's shares is 8. Common side effects from the drug include balance disorder and vomiting.

By late May, Sarepta had announced that the FDA wouldn't make a decision by the deadline and that it would work with the agency in its examination of the data.

"What she told them is you have to think about it, it to be in your calculus", said Ritu Baral, a senior biotech analyst at Cowen and Co.

The news comes following an historic turnout at the FDA's advisory committee hearing in April, which brought a record-breaking number of families, members of the medical community and supporters to Washington to testify on behalf of the DMD community in favor of treatment options for Duchenne.