The FDA, in its approval, also granted Sarepta a Pediatric Review Voucher to speed drug approval, as the company is developing follow-on drugs to Exondys 51. Executives said they anticipated health insurers would reimburse most patients for the cost, which they described as being in the middle of the typical price range for rare genetic disease treatments. Sidelining the protests from other senior members of the drug division, FDA Commissioner Dr. Robert M. Califf finally made a decision to provide the final approval late on Friday.
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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. “Not one drug we pushed for failed to make it through the clinical trial process”.
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. It is estimated that up to thirteen percent of people with DMD have mutations addressable by EXONDYS 51.
With BioMarin Pharmaceutical and PTC Therapeutics out of the way, Sarepta Therapeutics’ eteplirsen was the last drug standing. Previously there were no US –approved drugs to fight the disease, though steroid drugs have been used to slow the loss of muscle strength.
Shares of Sarepta, an OR company that moved to Cambridge in 2012 and now has about 230 employees, surged almost 74 percent to $48.94 on Monday. Until Monday, there was no approved therapy.
Sandy Walsh, an FDA spokeswoman, said Sarepta’s share price didn’t factor into Woodcock’s decision.
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.
Experts were divided on the clinical effectiveness of eteplirsen.
Sarepta’s stock was whipsawed in recent months amid speculation of an FDA rift.
“I think this is a collaborative effort that shows the FDA, companies and the patient community can work together toward a single goal, and that is improving the lives of patients”, said Furlong, founder and president of Parent Project Muscular Dystrophy, a nonprofit that helped fund travel and other expenses related to the study.
In April, the FDA held a a panel hearing during which impassioned patients and their caregivers wove stories of eteplirsen’s seemingly miraculous efficacy and pleaded for approval. Sarepta’s clinical studies supporting approval showed improvements in walking tests but involved just a dozen participants. The Company is focused on the discovery and development of ribonucleic acid (RNA)-targeted therapeutics for the treatment of rare, infectious and other diseases.
“We have written to Sarepta asking for an update on these plans, in light of the FDA approval”, adding that it is also trying to establish whether an orphan drug designation in the European Union for another exon 51-skipping therapy – BioMarin’s now-abandoned drisapersen – could block eteplirsen’s approval. McSherry, however, said her excitement was tempered by the knowledge that many other boys with Duchenne lost motor function during the FDA’s long delay.
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Im really overwhelmed, McLinn said.
