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PTC Gambles On Success With Emflaza; Says Price Revision Is Needed

 
• By Joseph Haas

Looking to become a therapeutic area leader in DMD, PTC pays $140m up front for Marathon's controversial Emflaza, but the biotech's stock price plummeted, indicating that investors don't share PTC's optimism.

group of businessman in black suit and holding red arrow down graph

PTC Therapeutics Inc., already at cross purposes with the US FDA over its re-filing of Translarna (ataluren) for nonsense-mutation Duchenne muscular dystrophy (DMD), apparently decided to take a chance on the drug-pricing controversy as well, paying $140m up front on March 16 to acquire all rights for Marathon Pharmaceuticals LLC's DMD drug Emflaza (deflazacort), the launch of which has been delayed by pricing pushback.

PTC execs said during a same-day investor call to outline the transaction and report fourth quarter earnings that the company plans to revise Marathon's initial Emflaza pricing of $89,000 per year, but they offered few specifics. Marathon attracted significant unwanted attention from Congress and the media with its planned pricing of the DMD drug, which has been available for decades overseas as a generic corticosteroid therapy

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