When PTC Therapeutics agreed to purchase Marathon's controversial drug Emflaza last week, it didn’t only get an FDA-approved treatment for Duchenne muscular dystrophy. It also inherited congressional scrutiny on the drug's price, originally set at a much-lambasted $89,000.
Pharma critics Sen. Bernie Sanders and Rep. Elijah Cummings are pressing the company for early pricing information on the beleaguered med, which has been under a cloud since its quick FDA approval last month. Its former owner, Marathon Pharma, attached that $89,000 price tag to a decades-old steroid drug that’s long been available in other countries for about $1,000 a year.
Facing a swell of backlash, Marathon pressed “pause” on the launch the same day Sanders and Cummings reached out to CEO Jeffrey Aronin for more information on the drug's development, costs and pricing decisions. A month later, Marathon sold Emflaza to PTC Therapeutics in a deal potentially worth more than $190 million.
Now, the lawmakers aren’t backing down just because Emflaza has a new owner. They’re urging PTC chief Stuart Peltz to “keep the price of this relatively common steroid at its current import cost.” They also want to learn whether PTC plans to seek approval in juvenile arthritis, an option they believed Marathon would pursue.
So far, PTC management has said it’s looking into Emflaza’s price and hasn’t decided on a final figure. The company has met with a patient group and plans to continue those conversations, according to the letter.
In a statement, Peltz said his company is “aware of the letter sent by Senator Sanders and Representative Cummings,” adding that the drugmaker plans “to respond within the designated timeframe.”
“As previously stated, PTC continues to engage with key stakeholders to understand how to make Emflaza available to patients who need it,” Peltz continued.
Writing on the deal last week, Barclays analysts said they “remain cautious” about the drug’s commercial potential in light of all the scrutiny.
Sanders and Cummings aren’t just going after industry in this instance, either. They’re asking the FDA to answer “serious questions” about the drug’s approval, among them whether the agency typically considers decades-old data and whether staff had concerns about the med.
Meanwhile, Marathon’s tactics with Emflaza have resulted in heightened scrutiny to U.S. orphan drug laws, a topic Sanders and Cummings hit in their letter. After picking up rights to old clinical data, the drugmaker was able to win orphan drug exclusivity for the med with relative ease. It also won a priority review voucher, which could be worth hundreds of millions of dollars in resale to another pharma company.
The U.S. Government Accountability Office this week said it plans to look into orphan drug laws by congressional request.
As a result of the Emflaza saga, industry group PhRMA said it’s reconsidering its membership criteria; Marathon is currently a member.