Marathon's $89,000 Drug Scrutinized for ‘Unusual’ ApprovalBy
Marathon Pharmaceuticals LLC is under new scrutiny as Washington lawmakers press U.S. regulators about the “unusual circumstances” surrounding approval of a drug that costs $89,000 a year.
Questions remain about the data used to evaluate Emflaza, a treatment for Duchenne muscular dystrophy, and why Marathon was given market exclusivity, according to a letter from two lawmakers to the U.S. Food and Drug Administration.
A generic form of Emflaza, called deflazacort, has been available to U.S. patients for decades from overseas pharmacies at a price of about $1,000. Closely held Marathon has become one of a string of U.S. drugmakers targeted for criticism after making quick, steep increases on the prices of older, widely available drugs.
“The high price was especially troubling in light of the incredibly lucrative benefits the FDA has granted to Marathon, and the limited amount of innovative research the company appears to have conducted to develop Emflaza,” Senator Bernie Sanders, an independent from Vermont, and Democratic Representative Elijah Cummings of Maryland said in a letter to FDA Acting Commissioner Stephen Ostroff.
FDA’s approval of the drug appears to have relied heavily on decades-old clinical trial data from a 1995 study, the lawmakers said in their letter.
Marathon spokeswoman Wanda Moebius didn’t immediately respond to a request for comment. FDA spokeswoman Sandy Walsh said the agency will review the letter and respond directly to Sanders and Cummings.
Sanders and Cummings sent a letter Feb. 13 to Marathon Chief Executive Officer Jeffrey Aronin requesting information about how much the company spent developing Emflaza. Northbrook, Illinois-based Marathon delayed the launch of Emflaza the same day.
Duchenne muscular dystrophy, or DMD, is a rare genetic disease that affects about one of every 3,600 male infants globally, according to the FDA. Patients lack a protein called dystrophin that’s essential for healthy muscle, and eventually lose the ability to walk, stand and breathe. Deflazacort is an anti-inflammatory corticosteriod used to treat the symptoms of DMD.