Passing the Emflaza Baton
Marathon sells Emflaza to PTC Therapeutics, but questions remain about FDA approval processes
The decision by Marathon Pharmaceuticals to charge $89,000 per patient per year for its Duchenne muscular dystrophy (DMD) drug, Emflaza (deflazacort), led to public outcry in February – and the company has quickly passed the baton, by selling the drug to PTC Therapeutics for $140 million. PTC paid upfront $75 million in cash and $65 million in stock – and Marathon is also set to receive royalties based on net sales (1).
But what happens to the price of Emflaza? Marathon landed itself in hot water after announcing the initial price, with Senator Bernie Sanders and Representative Elijah Cummings accusing the company of “abusing” the FDA’s orphan drug program. PTC has said it will “re-examine” the price tag but has yet to reveal any more specifics. PTC has already invested a significant amount of resources in DMD drug development. Its own DMD drug, Translarna, was approved in Europe in 2014 for nonsense mutation DMD, but has been denied FDA review on two occasions – although the drug was finally accepted for FDA review in early March.
Marathon was not the only one in the spotlight after announcing the $89,000 price tag – Sanders and Cummings have also raised questions about the FDA, including the data used to evaluate the drug and why “Marathon was given market exclusivity and other rewards for a 20-year-old drug that the company did no significant research on” (2).
Although deflazacort was only approved by the FDA in February, it has been available in other countries, including Canada and the UK, for many years – and has also been used off-label for DMD. A small number of US DMD patients had also been importing the drug, at a price tag of around $1200, according to some reports. In an effort to explain itself, Marathon wrote an open letter to the DMD community to describe the challenges of bringing the drug to market, but critics have pointed out that a lot of the development work was not performed by Marathon, since the company acquired the drug and rights to historical clinical data.
According to Sanders and Cummings, the high price of Emflaza was “especially troubling in light of the incredibly lucrative benefits FDA has granted to Marathon, and the limited amount of innovative research the company appears to have conducted to develop Emflaza”. As well as receiving orphan drug status, which grants 7 years of market exclusivity, Marathon has also received a priority review voucher, which it will be keeping after the sale.
In a letter to the FDA, Sanders and Cummings wrote, “FDA’s mission includes advancing public health by making medical products ‘more effective, safer, and more affordable.’ It is also the agency’s responsibility to ensure corporations are not gaming the system.”
- PTC Therapeutics, “PTC Therapeutics Announces Agreement to Acquire Emflaza for the Treatment of Duchenne Muscular Dystrophy in U.S,” (2017). Available at bit.ly/2nmAC8Y. Last accessed March 27, 2017.
- Committee on Oversight & Government Reform, “Sanders, Cummings Investigate Unusual Circumstances Surrounding FDA Approval of Muscular Dystrophy Drug,” (2017). Available at bit.ly/2nmFP0G. Last accessed March 27, 2017.
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