The agency OKed eteplirsen, which will be marketed as “Exondys 51”, through its accelerated approval pathway. Finally, WBB Securities reissued a “strong-buy” rating and issued a $60.00 price target (up from $40.00) on shares of Sarepta Therapeutics in a research note on Tuesday. About Exondys 51 Serepta’s Exondys 51 should be administered once a week at 30mg, once weekly. “We are grateful to the many patients and investigators who take partd in EXONDYS 51’s clinical studies”.
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People with DMD progressively lose the ability to perform activities independently and often require use of a wheelchair by their early teens. Patients typically die young so the agency’s decision was welcome news to families and patients who battle with the disease.
However, the FDA in its statement later added that, “a clinical benefit of Exondys 51, including improved motor function, has not been established”.
The accelerated approval was based on findings that the drug helped increase skeletal muscle in some patients, the FDA said. With the FDA nod, eteplirsen has become the first drug ever approved for Duchenne, a progressive and fatal genetic disease, and potentially has set a precedent for drug approvals in the U.S.
While shares of Sarepta have tumbled 26 percent lower this year-to-date, the biotech developer has two promising drugs in its development pipeline. DMD occurs in approximately one out of every 3,600 male infants worldwide. In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. This acknowledges that the patient voice is important, Furlong said.
“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. Its a really, really big day for the Duchenne community..
But Janet Woodcock, the head of the FDA’s Center for Drug Evaluation and Research, overruled the scientists’ objections and pushed for the drug’s approval, eventually backed by FDA commissioner of food and drugs Robert Califf.
Balance disorder and vomiting were the most common adverse events reported by participants treated with Exondys 51 in clinical trials.
Some health advocates criticized the FDAs decision.
Market Pulse Stories are Rapid-fire, short news bursts on stocks and markets as they move. Other drugs are now being developed for patients who have different mutations. Why would a company choose to do a careful, well-designed study that might show that its product isnt particularly safe or effective if it can get away with doing a tiny, poorly designed study with ambiguous results?. An estimated 13 percent of DMD patients have exon 51 skipping mutations.
“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.
Im really overwhelmed, McLinn said.
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But for now the FDA’s decision ends a years-long conflict between scientists and patients. 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.
