Sarepta Therapeutics' newly approved Duchenne muscular dystrophy drugeteplirsen will cost about $300,000 a year, depending on patient weight, according to STAT.
Edward Kaye, MD, CEO of Sarepta, said the drug's pricing was "in the middle of the range" for rare disease treatments. "And given the sensitivity to pricing, we tried to be reasonable when looking at all the costs," he told securities analysts in a phone call Monday.
Rare disease drugs — used to treat diseases with fewer than 200,000 patients in the U.S. — rose in average annual cost from $84,000 in 2010 to $112,000 in 2014, according to research firm EvaluatePharma.
"Considering the cost of manufacturing, delivering and supporting patients [with financial assistance programs], not to mention development costs over a decade, I don't think it's too much," Debra Miller, cofounder of CureDuchenne, an advocacy group that invests in drug makers, said of eteplirsen's cost.
The FDA deferred its decision on whether to approve eteplirsen in late May after an advisory panel concluded Sarepta's 12-patient clinical trial did not prove the drug's effectiveness. The deferral led to an intense and emotional public campaign from patients, parents and physicians calling for the drug's approval. Eteplirsen's ultimate approval stemmed from a very contentious internal review process, which involved contradictory opinions from FDA experts and executives, reported The Wall Street Journal.
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