Drugmaker to sell muscular dystrophy drug amid pricing backlash

PTC Therapeutics on Thursday announced plans to buy and review the price of Marathon Pharmaceuticals' recently-approved drug to treat Duchenne muscular dystrophy.

Background

FDA in February approved Marathon's deflazacort, known under the brand name Emflaza, for sale in the United States to treat Duchenne muscular dystrophy. FDA also granted Emflaza Orphan drug designation, meaning FDA will not approve another version of the drug during that seven-year period even if the company's patent expires.

Following the drug's approval, Marathon announced that it would set Emflaza's list price at $89,000 for a year's supply. Marathon CFO Babar Ghias had said the company planned to begin selling the drug in March.

However, stakeholders immediately criticized Marathon's proposed list price for Emflaza. Amid the backlash, Marathon CEO Jeffrey Aronin announced that the company would delay the drug's launch.

PTC to buy Emflaza

PTC said it has reached a deal with Marathon to purchase Emflaza for $140 million. Marathon will receive $75 million in cash and $66 million in PTC stock for the drug. Marathon also will be eligible to receive a percentage of the drug's sales starting in 2018, as well as a one-time $50 million payment when Emflaza reaches an undisclosed sales-based milestone, Kaiser Health News reports.

PTC CEO Stuart Peltz said PTC executives expect 9,000 U.S. patients to use the Emflaza. PTC did not announce details on how it will price Emflaza, but the company said it will review the drug's price. "We're re-examining the current price of Emflaza ... We believe a change needs to be had, but ... it's really too premature to speculate on the price level," Peltz said.

Peltz added that PTC plans to reach out to the Duchenne community, physicians, and payers about the drug's price and announce pricing details after the purchase is finalized, which is expected to occur by the end of June (Grover, Reuters, 3/16; Tribble, Kaiser Health News, 3/16; Walker/Steele, Wall Street Journal, 3/16).

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