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Biotech stock explodes 80% after the FDA approves a key drug

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There are now no drugs approved in the United States to directly treat any of these groups.

The new drug targets a genetic mutation that affects about 13 percent of Duchenne's patients.

DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. Ellis Unger, the FDA executive who oversaw the review, said in documents calling for the treatment to be rejected that there was no consensus on whether the small amounts of the protein that Sarepta's drug helped Duchenne patients produce were enough to improve their condition. The disease slowly destroys patients' muscles and results in death, often by age 30. The highest share price NASDAQ:SRPT shares have reached in the prior twelve months is 56.18 with the lowest being 8.

Sarepta Therapeutics says it may be 60 to 90 days before it begins generating revenue from Exondys 51, so it will still be a bit before we fully understand the commercial opportunity for this drug.

These include the requirement that Sarepta conduct a further clinical trial to confirm Exondys 51's clinical benefit, assessing how well Exondys 51 improves motor function in DMD patients with a dystrophin mutation gene amenable to exon 51 skipping.

Some staffers said there was little evidence the drug worked. That advisory vote was met with significant pushback from patients' families, some physicians and politicians, the Associated Press reported. But when it comes to formal approval, it's the job of FDA officials to give the green light only to proven therapies, without being distracted by emotions. Exondys 51 will cost around $300,000 annually for patients with Duchenne muscular dystrophy. If the study fails to shows it helps, the FDA said it could withdraw the drug.

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Pat Furlong, a patient advocate who lost two sons to the disease, called the announcement 'an extraordinary win'.

The U.S. National Institutes of Health provides more information on Duchenne muscular dystrophy. Much of the sellside had written off its chances of approval - with good reason, as the FDA rarely disagrees with a negative adcom vote - so approval came as a surprise, and a huge spike in the share price was an obvious outcome.

At a public meeting in April, an outside panel of experts did not support the drug's approval, voting 7-3 that it did not show effectiveness in treating the disease.

However, the FDA's approval of the drug is "contentious", the firm noted, and the efficacy of Exondys is still "questionable". Did the FDA make the right decision? In an August 8 memo to Unger, Dr. Luciana Borio, the FDA acting chief scientist who convened the board that reviewed the dispute, wrote that "we fear that those actions could have chilled scientific debate within (the FDA Center for Drug Evaluation and Review) and reduced the level of participation by the review team during the final stages of the decision-making process". And not just because they hope to keep taking the drug. The Company is also developing therapeutics using its technology for the treatment of drug-resistant bacteria and infectious, rare and other human diseases.

Shares of Sarepta Therapeutics rose more than 73 per cent Monday to close at $48.94 in regular trading.

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