A drug sold for years overseas to treat a rare form of muscular dystrophy was OKed Thursday by the FDA to be sold in the US, but it comes at a high price. The Wall Street Journal reports the now-FDA-approved deflazacort will be offered by Marathon Pharmaceuticals for $89,000 or so a year, up to 70 times its cost outside the US; Marathon CFO Babar Ghias tells the Washington Post the net price will be $54,000 after rebates and discounts. Some who rely on it to treat Duchenne muscular dystrophy—an incurable disease most often affecting boys and typically leading to death by one's 30s—are afraid they won't be able to afford it if insurance won't pay for it or if deductibles are too high. The drug, which Ghias tells the Journal will be sold under the brand Emflaza starting in March, is a steroid that helps mitigate muscle weakness and cut inflammation, per an FDA release.
Emflaza is also said to cause fewer side effects than prednisone, a non-FDA-approved drug often prescribed for DMD, per Reuters. Marathon had to invest money in new trials before the FDA would approve US sales. And, like other pharma companies that scoop up "orphan drugs"—meds used for diseases that don't affect more than 200,000 people nationwide—Marathon says the high cost is justified because of the money it has poured into R&D. A Massachusetts woman who's been buying the drug for her son for $1,600 a year from a UK online pharmacy isn't seeing it the same way, telling the Journal she's "disappointed" Marathon boosted her cost "by more than $87,000 a year." Because the FDA has approved a drug to treat the disease, US consumers won't be legally able to acquire it overseas at the cheaper price anymore. (A pharma company that's been labeled a major "tax dodger.")