Under the provisions of the accelerated approval process, the FDA is requiring Sarepta Therapeutics to conduct a clinical trial to confirm the drug’s clinical benefit, specifically determining whether eteplirsen improves motor function in patients with the disease who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping.
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Documents published by the FDA reviewers ahead of an advisory committee meeting raised major doubts about its efficacy, and those concerns were played out at the meeting with only three of 10 panellists voting that the drug is effective in DMD.
They had been working with United States biotechnology company Sarepta Therapeutics to get the drug through clinical trials.
Only yesterday Sarepta Therapeutics Inc (NASDAQ:SRPT) traded 91.30% higher at $51.30. About Exondys 51 Serepta’s Exondys 51 should be administered once a week at 30mg, once weekly.
Federal regulators have granted tentative approval to the first drug for muscular dystrophy – despite little evidence to prove it works. It is designed for the 13 percent of patients with a particular genetic mutation that prevents them from making dystrophin, a key protein that keeps muscles intact. The condition nearly exclusively affects boys and leaves many dead by their mid-20s. Patients typically succumb to the disease in their 20s or 30s; however, disease severity and life expectancy vary.
In response to the news, Sarepta announced that it will launching Exondys 51 for sale “immediately”.
The accelerated approval of Exondys 51 is based on the surrogate endpoint of dystrophin increase in skeletal muscle observed in some Exondys 51-treated patients.
In the meantime Sarepta has said it will move swiftly to launch the drug, which has been given a list price of $300,000 a year. If the drug doesnt help, the FDA could withdraw approval.
In terms of Buy/Sell recommendations, analysts have a consensus rating of 1.93 on the shares of Sarepta Therapeutics Inc (NASDAQ:SRPT). “If the drug lives up to its expectations I think (the company) gets taken out”, said Debjit Chattopadhyay, managing director at Janney Montgomery Scott, which has a “buy” rating on the stock.
Farkas had expressed doubts about the drugs effectiveness during a review of Exondys 51 earlier this year. It occurs in about one out of every 3,600 male infants worldwide.
A new drug for the treatment of a rare and fatal muscle wasting disease, developed by West Australian researchers, has been approved by the US Food and Drug Administration.
“By allowing the marketing of an ineffective drug, essentially a scientifically elegant placebo, thousands of patients and their families would be given false hope in exchange for hardship and risk”, said Unger.
In the meantime, though, Simeonidis says the approval, which has been very much a question, makes it “very likely that Sarepta will be acquired for eteplirsen and its pipeline well before data” from the two-year study are required. This acknowledges that the patient voice is important, Furlong said. Many patients must use a wheelchair by the time they are teenagers. Its a really, really big day for the Duchenne community.. The most frequently reported adverse effects of the drug include balance disorder and vomiting.
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Some health advocates criticized the FDAs decision.
