Marathon Pharmaceuticals has paused the commercial launch of Emflaza (deflazacort) – approved by the FDA on February 9, 2017, for treating Duchenne muscular dystrophy (DMD) – after public outcry over the $89,000 price tag per patient per year (1). Marathon describe Emflaza as the “first widely-applicable treatment for the rare fatal genetic disorder” – and the drug received priority review under the FDA’s orphan drug program (2). Critics, however, claim that deflazacort is not technically a new drug – it has been approved in Europe for years and used off-label for DMD, along with other steroids where the benefits are well documented. A small number of US DMD patients have also been importing deflazacort at a cost of around $1000 per year. Until now, the drug has never been approved by the FDA for any indication so Emflaza has followed the path (and costs) of an entirely new drug in the US. Marathon decided to pursue development after acquiring historical data that showed a potential benefit in DMD.
In an open letter to the Duchenne community, Marathon wrote, “The resources we invested were substantial and we don’t expect to recoup our investment for several years and we have only 7 years of market exclusivity.” (1) The company also emphasizes that deflazacort has never been approved anywhere in the world for Duchenne, even though it was frequently prescribed off label. “Our goal in commercializing EMFLAZA all along has been to make it available to a much broader set of patients who, prior to FDA approval, have not had access to this therapy. There was also much we did not know about using the drug in the Duchenne patient population including proper dosing, potential side effects and drug-to-drug interactions,” explained Marathon’s CEO, Jeff Aronin. Many people, however, seem unconvinced by the argument. Senator Bernie Sanders and Representative Elijah Cummings have also gotten involved by sending a letter to Marathon’s CEO demanding an explanation for the high price – and also accusing the company of abusing the FDA’s orphan drug development program. “We believe Marathon is abusing our nation’s 'orphan drug' program, which grants companies seven years of market exclusivity to encourage research into new treatments for rare diseases – not to provide companies like Marathon with lucrative market exclusivity rights for drugs that have been available for decades,” Sanders and Cummings wrote (3).
Critics have also pointed out that although deflazacort can help DMD patients by boosting muscle strength and physical function, it is not a cure: DMD is ultimately still fatal, with the majority of patients only living to their thirties. It’s not yet known when the commercial rollout of deflazacort in the US will begin – Marathon says it will be meeting with Duchenne community leaders to explain the commercialization plans and discuss options. In the meantime, the media storm continues to grow. In the latest development, rumors are also abound that Pharmaceutical Research and Manufacturers of America (PhRMA) is considering action. PhRMA is reportedly reviewing its membership criteria, adding that Marathon’s “recent actions are not consistent with the mission of our organization”. (4)
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References
- Marathon Pharmaceuticals, “An Open Letter to the Duchenne Community,” (2017). Available at: http://bit.ly/2lfVa3Z. Last accessed February 20, 2017. Marathon Pharmaceuticals, “EMFLAZA (deflazacort) has just been FDA-approved,” (2017). Available at: http://bit.ly/2ly87no. Last accessed February 20, 2017. The Washington Post, “Lawmakers slam new $89,000 price tag on rare disease treatment: ‘Unconscionable’,” (2017). Available at: http://wapo.st/2kE2Vli. Last accessed February 20, 2017. Bloomberg, “Pharma Lobby May Boot Company That Introduced High-Priced Drug,” (2017). Available at: http://bloom.bg/2kUfcAD. Last accessed February 20, 2017.
