The clinical operations will need to confirm that Exondys indeed does more than improve biomarkers in patients, and actually changes patient outcomes.
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“Earlier this year, Sarepta Therapeutics told Muscular Dystrophy UK that they plan to apply to the EMA by the end of 2016”, said the charity in a statement. However, during the drug’s FDA advisory committee review in April, the panel of experts voted against recommending an approval by a margin of 7 to 6.
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.
“These kids have hope now”, said Christine McSherry of Pembroke, whose 20-year-old son Jett benefitted from Sarepta’s treatment, called eteplirsen, in the clinical trial. The approval is still conditional since the basis was predominantly from a study conducted, which involved only 12 boys. Unger warned that it would lower the agency’s standards and establish a precedent that regulators could be forced to bend in the face of intensive PR campaigns by patients and advocates, even though the FDA is supposed to hew to the empirical evidence. While the decision delighted the advocates of the drug who actively lobbied for its approval for years, drug policy experts are anxious about FDA’s decision.
“We had the feeling we were screaming for the life of these boys and no one could hear us”, said Debra Miller, founder of CureDuchenne of Newport Beach, Calif., one of three patient groups that helped fund Sarepta’s clinical trial. Exondys 51 would treat about 13 percent of those boys, a subset with a gene mutation that makes them unable to produce a protein called dystrophin that can help bolster motor function.
“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease”, said Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research. The annual cost for an average size child could reach highs of $300,000 according to initial estimates. Until Monday, there was no approved therapy.
There was also internal debate at the agency over whether the drug’s effect on protein production was enough to approve a medication. The company is based in Cambridge, Massachusetts.
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As always, accelerated approval came with significant post-approval obligations.
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The approval decision is far from unanimous however, with Woodcock in opposition to fellow CDER officials including CDER deputy director Ellis Unger who appealed the decision to approve via the FDA’s internal disputes procedures. The Company is also developing therapeutics using its technology for the treatment of drug-resistant bacteria and infectious, rare and other human diseases. The decision may result in negative consequences for the accelerated review of such a rare disease drug, said Mr. Simeonidis.
