Sanders, Cummings Investigate Unusual Circumstances Surrounding FDA Approval of Muscular Dystrophy Drug

Sanders, Cummings Investigate Unusual Circumstances Surrounding FDA Approval of Muscular Dystrophy Drug

WASHINGTON, March 15 – Sen. Bernie Sanders (I-Vt.) and Rep. Elijah E. Cummings (D-Md.), ranking member of the House Committee on Oversight and Government Reform, sent a letter to the Food and Drug Administration Wednesday requesting information about the unusual circumstances surrounding the approval of Emflaza – a brand name version of a drug used to treat a deadly genetic muscle deterioration disorder.

Marathon Pharmaceuticals, the company that will market Emflaza, backtracked on plans to sell the drug for the obscenely high price of $89,000 a year after Sanders and Cummings sent a letter to the company criticizing the cost. However, questions remain about the data FDA used to evaluate the drug and why Marathon was given market exclusivity and other rewards for a 20-year-old drug that the company did no significant research on.

"The high price was especially troubling in light of the incredibly lucrative benefits FDA has granted to Marathon, and the limited amount of innovative research the company appears to have conducted to develop Emflaza," Sanders and Cummings wrote in a letter to FDA Acting Commissioner Dr. Stephen Ostroff.

Among those lucrative benefits was the agency's designation of Emflaza as an orphan drug, thereby granting Marathon a monopoly on the market for seven years. Furthermore, even though the drug is decades old, Marathon received a valuable pediatric priority review voucher (PRV), worth potentially hundreds of millions of dollars, to use or sell to another pharmaceutical company

"FDA's mission includes advancing public health by making medical products ‘more effective, safer, and more affordable.' It is also the agency's responsibility to ensure corporations are not gaming the system," Sanders and Cummings wrote.

A review of documents, Sanders and Cummings said, "raised serious questions about FDA's decision regarding Emflaza."

To read the letter, click here.