March 16 (Reuters) - Marathon Pharmaceuticals LLC, under intense pressure from U.S. lawmakers for the hefty price tag on its Duchenne muscular dystrophy drug, has agreed to sell the rights to the recently-approved treatment to PTC Therapeutics Inc.
PTC said on Thursday it would buy the rights to the steroid, Emflaza, from Marathon for $140 million upfront, comprised of about $75 million in cash and about $65 million in stock.
Emflaza has been available outside the United States for decades to treat DMD, a devastating muscle-wasting disease that mainly affects young boys. Last month, the U.S. Food and Drug Administration approved Emflaza for use in the United States.
But, Marathon halted the drug’s launch after lawmakers questioned the company pricing the drug at $89,000 per year when patients had been able to import it for as little as $1,000.
PTC, which has been involved with developing DMD treatments for nearly 20 years, sells its own DMD drug, Translarna, in Europe.
But Translarna, which is designed to treat a specific subset of DMD patients, was rejected by the FDA last year and is currently being reviewed for use in the United States.
The only other U.S.-approved DMD drug is Sarepta Therapeutics’ Exondys 51, which was cleared in September despite an outside panel of experts and the FDA’s own reviewers questioning its efficacy.
Until then, there were no specific treatments in the United States for the rare, progressive genetic disorder that hampers muscle movement, eventually killing most sufferers by age 30.
PTC said Marathon is also eligible to receive certain royalties and milestone payments. The deal is expected to close in the second quarter. (Reporting by Natalie Grover in Bengaluru; Editing by Savio D‘Souza)