Company Delays Launch of Muscular Dystrophy Drug

Feb. 14, 2017 -- The U.S. launch of a drug for a rare disorder called Duchenne muscular dystrophy will be paused due to concerns about its price, Marathon Pharmaceuticals told patient advocates this week.

After its drug Emflaza was approved last week by the Food and Drug Administration, the company said it would cost $89,000 a year, which resulted in a barrage of criticism from patients and lawmakers, Kaiser Health News reported.

American patients and their families have been importing a generic version of the drug deflazocort for about $1,200 a year.

"Our goal in commercializing Emflaza all along has been to make it available to that broader set of patients who prior to FDA approval have not had access to the therapy We are pausing our launch, which has not yet taken place. We have not sold any new product and will pause that process," said a statement attributed to Marathon CEO Jeffrey Aronin that was read to a conference of parents, patients and advocates in Washington, D.C., Kaiser reported.

Marathon will continue to offer patients an expanded access program, which allows about 800 patients to receive the drug from the company, according to the statement.

It also said that more patients can join that program for free, and patients and their families can continue importing the generic version, Kaiser reported.

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