BioMarin Pharmaceutical Inc said on Thursday the U.S. Food and Drug Administration had rejected the company’s drug to treat a rare muscle-wasting disorder.
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Reviewers who weighed in on eteplirsen wrote that “the data overall did not provide statistical evidence to support the efficacy” in Duchenne patients. Mostly DMD patients lose their battle against the disease by the age of 30.
Sarepta is developing its own treatment for DMD, a drug called eteplirsen, which will face an FDA review on Friday January 22. Brozak added that the pressure for the treatment approval is going to mount. Sarepta’s eteplirsen, like BioMarin’s Kyndrisa, has skipped a defective part of the gene for the production of dystrophin, the lack of which has been believed to have resulted into DMD.
Heather Behanna, an analyst from Wedbush Securities, said that the two drugs could benefit a certain group of people with DMD accounting to about 1,800 boys in the United States and another 5,000 around the globe. That buyout deal included a $160 million set of milestones for an early approval of drisa, which will now be seriously delayed before a return trip to US regulators.
While a US approval would have given BioMarin first-mover advantage in the market, a European approval is perhaps more significant as the drug will target a larger patient population. However, the FDA’s briefing documents seem to indicate otherwise, meaning that it’s looking like Sarepta will ultimately have to complete a late-stage study to clear up any outstanding questions regarding eteplirsen’s effectiveness. The agency’s rejection has shifted investor focus to Sarepta Therapeutics Inc’s rival drug, which is presently in development.
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Shares traded recently at $15.16, down 52 percent. Sarepta stock followed a similar course but was down 6% midday.
