$89,000 Drug For Muscular Dystrophy Dropped By Marathon

NORTHBROOK, IL — A day after members of Congress sent a second letter questioning its prices, Marathon Pharmaceuticals announced it is getting rid of the muscular dystrophy drug it proposed selling for $89,000.

The hefty price tag caught extra attention because a generic version (deflazacort) of the same treatment had been available for import for around $1,500 before Marathon obtained FDA approval and exclusive rights to sell the drug under the name Emflaza.

After public outcry, Marathon CEO Jeffrey Aronin announced last month the company was putting the planned price hike for the drug on hold. Now, it plans to offload it permanently, the company revealed Thursday.

Northbrook-based Marathon will sell Emflaza to PTC Therapeutrics of South Plainfield, New Jersey for $140 million ($75 million in cash and approximately $65 million in PTC stock) and a possible bonus payment of $50 million is the drug meets its sales goals. The payments will begin in 2018, according to the announcement from PTC.

Emflaza treats Duchenne muscular dystrophy (DMD,) a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death due to heart and respiratory failure. The head of PTC says the acquisition of the drug fits into the company's mission to bring treatments to those suffering from rare diseases.

» Marathon Pharmaceuticals "pauses" release of $89,000 drug

"With our nearly 20-year commitment to the Duchenne community, it is deeply meaningful for us to bring this critical therapy to U.S. patients," said Stuart W. Peltz, Ph.D., CEO of PTC Therapeutics, Inc. "We believe Emflaza is a disease-modifying therapy that has been shown to slow disease progression. In keeping with PTC's mission, we are excited to work with the community to raise the standard of care for DMD patients."

» Generic drug gets 6,000 percent price increase

Representatives from PTC on the conference call announcing the sale said they plan to "reexamine the price," of the drug but did not specify how much they planned to charge for the drug, according to the Chicago Tribune.

Two liberal lawmakers, Sen. Bernie Sanders (I, VT) and Rep. Elijah Cummings (D, MD-7) sent a letter to the Food and Drug Administration Wednesday about what they described as "unusual circumstances" around the FDA's approval of Emflaza last month.

Sanders and Cummings said they found the price "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," the Tribune reported.