CHICAGO _ Marathon Pharmaceuticals is selling its $89,000 muscular dystrophy drug, the company announced Thursday morning.
Marathon, headquartered in suburban Chicago, will sell the drug to PTC Therapeutics, a South Plainfield, N.J., company for $140 million in cash and stock upfront. Marathon also will receive payments from PTC based on the sale of the drug, Emflaza, starting in 2018. Additionally, Marathon may receive a one-time $50 million payment if the drug reaches a sales-based milestone.
Emflaza was just approved by the U.S. Food and Drug Administration last month to treat a fatal muscle wasting disease called Duchenne muscular dystrophy. Marathon caused an uproar when it said it would charge a list price of $89,000 a year for the drug, which has been available outside the U.S. for years for as little as $1,200 a year.
Marathon posted a letter on its website Thursday, saying, "We believe this transaction will help create the opportunities needed to ensure the greatest number of patients with Duchenne who might benefit from EMFLAZA can receive this drug." The letter praised PTC's "deep-rooted experience with Duchenne."
When asked if the pricing controversy is what drove Marathon to sell the drug, a Marathon spokeswoman pointed back to that letter posted on the company's website.
PTC leaders did not say how much they planned to charge for Emflaza on a call with investors Thursday. Attempts to reach PTC for comment were not immediately successful. But PTC Chief Commercial Officer Mark Rothera said on the call that "we plan to reexamine the price of Emflaza and work with key stakeholders to ensure patient access is a priority."
PTC also plans to retain "certain key members of the Marathon team who are expected to pay a critical role in the launch," PTC Chief Financial Officer Shane Kovacs said in the call.
Rothera said PTC is still finalizing its plans to sell the drug and will provide more information after the transaction closes. The sale is expected to close in the second quarter of this year.
PTC already sells a different Duchenne drug, called Translarna, outside the United States. The FDA has not yet approved Translarna for sale here. Last year, the FDA told PTC that its application for approval of Translarna was not complete enough to be reviewed. Translarna targets only about 13 percent of Duchenne patients, those with a specific genetic mutation, whereas Emflaza can be used much more widely.
Rothera noted that launching Emflaza will allow PTC to establish "a complete U.S. sales and marketing footprint ahead of a possible Translarna launch."
