Marathon's $89,000 Drug May Exploit Patients, Senators Say

(Bloomberg) -- Marathon Pharmaceuticals LLC is facing more criticism over the $89,000 price tag on its drug for a rare muscle disorder as a group of eight U.S. lawmakers said they're concerned that it “exploits” patients.

In a March 3 letter to Marathon Chief Executive Officer Jeffrey Aronin, seven Democratic U.S. senators and one independent demanded information on the closely held drugmaker's pricing practices and product development costs for Emflaza, used to treat lethal Duchenne muscular dystrophy.

The letter adds to the outcry that started last month when Marathon first announced the drug's price, prompting the drugmaker to delay Emflaza's introduction. The backlash was exacerbated by the fact that U.S. patients have been able to buy a generic version of Marathon's drug from overseas pharmacies for about $1,000 for decades.

“We are concerned that Marathon's pricing unfairly exploits the DMD patient population,” the senators said in their letter.

Marathon is committed to ensuring that all patients who need the drug have access to it, spokeswoman Wanda Moebius said in an emailed statement. “During this pause in commercialization we continue to work with stakeholders in the Duchenne muscular dystrophy community on the best path forward to accomplish that important goal.”

When the drug was approved the U.S. last month, the company said it had conducted 17 preclinical and clinical studies to support its application.

Earlier Letter

Companies including Mylan NV and Valeant Pharmaceuticals International Inc. have been scrutinized by Washington lawmakers and federal agencies for quickly raising prices on relatively cheap drugs, and President Donald Trump has accused the industry of “getting away with murder.” 

The March 3 letter was signed by Democratic senators Patty Murray of Washington, Tammy Baldwin of Wisconsin, Cory Booker of New Jersey, Al Franken of Minnesota, Maggie Hassan of New Hampshire, Elizabeth Warren of Massachusetts, Sheldon Whitehouse of Rhode Island, and independent Angus King of Maine. 

Marathon paused the launch of Emflaza on Feb. 13, days after getting clearance in the U.S. Senator Bernie Sanders and Representative Elijah Cummings also sent a letter to Aronin, calling its price “outrageous.”

DMD is a rare genetic disease that affects about one of every 3,600 male infants globally. Patients lack a protein called dystrophin that's essential for muscle health, and eventually lose the ability to walk, stand and breathe.

Emflaza, an anti-inflammatory corticosteroid, is used to treat the symptoms of DMD. About 22 percent of patients take the generic version, called deflazacort, according to the senator's letter. Deflazacort isn't approved for sale in the U.S.

Emflaza is the second treatment to be approved in the U.S. for the disease. The first, Sarepta Therapeutics Inc.'s Exondys 51, is designed to treat the disease's underlying cause by helping patients with a certain genetic profile make dystrophin. The letter praised Sarepta as an example of development costs justifying the eventual price.

To contact the reporter on this story: Katherine Greifeld in New York at kgreifeld@bloomberg.net.

To contact the editors responsible for this story: Drew Armstrong at darmstrong17@bloomberg.net, John Lauerman, Cecile Daurat