US lawmakers question price of Marathon's DMD drug Emflaza; drugmaker "pausing" launch

US Representative Elijah Cummings and Senator Bernie Sanders on Monday asked Marathon Pharmaceuticals to justify the list price of the recently approved Duchenne muscular dystrophy (DMD) drug Emflaza (deflazacort). "We urge you to significantly lower your price for this drug before it goes on the market next month," Cummings and Sanders remarked, adding "Marathon's apparent abuse of government-granted exclusivity periods and incentives to sell what should be a widely available drug for $89 000 a year is unconscionable." 

Specifically, the lawmakers called upon Marathon to clarify the cost of development of Emflaza as well as the drug's acquisition history. Cummings and Sanders additionally directed the drugmaker to disclose how the price was set and how much the company expects to generate in revenue from the drug. 

In their communication, the lawmakers accused Marathon of taking advantage of the orphan drug programme for a drug they said it did not develop, stating "Marathon acquired the rights to historical clinical trial data from the 1990s and completed some additional analyses to gain approval from the [FDA] to sell the drug in the US." Cummings and Sanders explained that the programme was not intended to "provide companies like Marathon with lucrative market exclusivity rights for drugs that have been available for decades." 

Marathon has estimated that the net price of Emflaza in the US following discounts and rebates will be $54 000 a year, while chief financial officer Babar Ghias has indicated that uninsured patients will be able to obtain the drug at no cost through a company programme.  Cummings and Sanders noted that the therapy is available in other countries such as Canada and the UK for about $1000 a year, adding that patients have imported the therapy to the US for "decades." 

Meanwhile, Marathon CEO Jeffrey Aronin said Monday that the company will pause the launch of Emflaza "for an unspecified amount of time", following discussions with patients and advocacy groups regarding how pricing and reimbursement decisions could affect DMD treatment, noting that the company "will meet with caregivers and explain our commercialisation plans, review their concerns, discuss all options, and move forward with commercialisation based on an agreed plan of action." Aronin added "we will maintain our expanded access programme, through which 800 patients currently receive Emflaza free of charge," continuing "if new patients seek to be included in our [programme], we will make that possible for free." 

In 2014, Cummings and Sanders directed Marathon to explain price increases on the cardiovascular therapies Isuprel (isoproterenol) and Nitropress (nitroprusside). The drugs were sold to Valeant Pharmaceuticals the following year. 

The news comes after the Pharmaceutical Research and Manufacturers of America announced plans to launch an advertising campaign amid criticism over pricing practices in the industry. Earlier this year, US President Donald Trump accused drugmakers of "getting away with murder" and vowed to implement a bidding process for government business. 

For related analysis, read ViewPoints: Marathon reveals approval plans for ancient corticosteroid in DMD – pricing will be under a microscope. See also, Spotlight On: Diversification, M&A and continued US pricing pressure likely to shape pharma in 2017, argue industry executives.