March 16 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
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
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