The US Food and Drug Administration has granted a conditional approval to Sarepta’s Exondys 51, the first drug cleared to treat patients with Duchenne muscular dystrophy (DMD).
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The Murdoch University scientists developed an innovative treatment to help sufferers of Duchenne muscular dystrophy, a crippling muscle-wasting disease that affects about one in 3 500 boys worldwide.
Sarepta Therapeutics Inc (NASDAQ:SRPT) has already started proceedings to market the drug, reiterating that the drug’s costs will be based on a patient’s weight.
Sarepta Therapeutics (NASDAQ:SRPT) opened at 48.94 on Monday. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
While noting that the clinical benefit of Exondys 51, including improved motor function, has not yet been established, the FDA said it considered the potential risks associated with the drug, and balanced them against the life-threatening and debilitating nature of the disease and the lack of alternate available therapies. The FDA encourages companies to develop drugs for orphan diseases by giving them special tax credits and extending the amount of time that companies are able to sell them exclusively, without generic competition.
Sarepta has become the first company to get U.S. approval for a drug to treat Duchenne muscular dystrophy, with the FDA overruling its own advisors in backing the drug. He had always defended himself by stating that he was trying to analyze the drug from multiple viewpoints rather than disabling the patients from getting the required treatment. Needham & Company LLC increased their price objective on Sarepta Therapeutics from $47.00 to $81.00 and gave the company a “buy” rating in a research report on Tuesday.
The disease is so rare that the FDA considers it an orphan disease, or one that isnt common enough to attract many drug developers. This indication is based on a boost in dystrophin in skeletal muscles observed in some patients treated with EXONDYS 51.
Farkas had expressed doubts about the drugs effectiveness during a review of Exondys 51 earlier this year.
Under the accelerated approval provisions, the FDA is requiring Sarepta to conduct a study to confirm the drug’s clinical benefit.
The approval went against the recommendation of experts who did not find enough evidence that the drug was effective.
The breakthrough was reached when Dr. Janet Woodcock made a decision to approve the eteplirsen drug, citing community needs.
According to agency memos posted to the FDA’s website after the announcement, Califf said that he would defer to Woodcock’s “judgment and authority” to approve the drug, despite opposition by several other agency staffers.
That the drug appears to have few side effects also likely helped with the decision, said Sam Fazeli, an analyst with Bloomberg Intelligence, as did the fact the patients had no other options. “Not one drug we pushed for failed to make it through the clinical trial process”.
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“It’s the first treatment for DMD that addresses the cause of the disease and the hope is that it will slow its progression and keep patients mobile for longer”, Professor Owens said. Priority review status is granted to applications for drugs that, if approved, would be a significant improvement in safety or effectiveness in the treatment of a serious condition.
