Brief

Marathon presses pause on Emflaza launch after pricing pushback

Dive Brief:

  • Marathon Pharmaceuticals announced Monday it would pause commercialization of its newly approved treatment for Duchenne muscular dystrophy, responding to widespread criticism of its decision to price the drug at $89,000 per year. 
  • In a letter to patients, Marathon CEO Jeff Aronin said the company would meet with community leaders to hear out their concerns and review options for marketing Emflaza (deflazacort), an old steroid which had been used off-label to treat DMD for years. 
  • The controversy over Marathon's pricing of Emflaza has echoes of previous pricing firestorms which have sparked scrutiny over industry practices and incentives. 

Dive Insight:

A sharply critical letter sent to the company from prominent Democratic Senators Bernie Sanders, I-VT, and Elijah Cummings, D-MD. seemed to trigger Marathon's step back from its commercialization plans.

Sanders and Cummings accused Marathon of abusing the orphan drug program, under which Marathon received seven years of market exclusivity for securing approval of Emflaza for DMD — a relatively rare muscle-wasting disease.

Emflaza has been available overseas as a generic corticosteroid for years, but was never approved in the U.S or for use in DMD patients in any regulatory jurisdiction. 

In order to get the drug approved for DMD in the U.S., Marathon acquired the rights to decades-old data from two efficacy studies of deflazacort. The Illinois-based drugmaker then carried out an array of clinical and preclinical studies investigating toxicity, drug-drug interactions and other characteristics, but didn't conduct any new pivotal trials.

The lawmakers asked Marathon to provide its total expenses for developing and securing U.S. approval of the drug, as well as its sales and profit projections.

Sanders and Cummings didn't mince words about their purpose in writing, either: "We urge you to significantly lower your price for this before it goes on the market next month. 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." 

Marathon has said the net price will be substantially below that $89,000 list price after rebates and discounts given to payers are taken into account. Company CEO Aronin has also insisted Marathon won't recoup its investment on developing Emflaza for several years, and emphasized most patients would pay a co-pay below $20 per prescription. 

After the likes of Turing, Valeant and Mylan, this may all seem like deja vu. But there are some reasons why this controversy may come to matter more than another example of pharma tone-deafness. 

Marathon Pharmaceuticals is a member of the powerful trade association PhRMA, which just rolled out a shiny new marketing campaign aimed at emphasizing the work of pioneering scientists striving to "cure the incurable."

PhRMA, and many major pharmas, have sought to distance themselves from "bad actors" in the industry, drawing a line between innovative companies and those that repurpose old drugs to raise prices. In a similar vein, Allergan, AbbVie, Takeda and Novo Nordisk have all pledged in recent months to keep annual price increases to single digits - although AbbVie only indicated it would do so for this year. 

The outcry over Marathon could intensify the pressure on larger pharma companies to differentiate themselves, perhaps driving more to take public stances on pricing. 

Marathon's case might also serve as fuel for lawmakers interested in reworking regulatory incentives, such as the orphan drug program. Republican Senator Chuck Grassley, R-IA, for example, has said he will investigate possible misuse of the program. And on Tuesday, he released a letter with Senators John McCain, R-AZ, and Amy Klobuchar, D-MN, urging the Department of Health and Human Services to consider importation of prescription drugs from Canada. 

While Trump may have taken a step back from his more strident criticism of the industry in recent weeks, pharma may still be in for a roller-coaster ride in the coming years.

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Filed Under: Regulatory / Compliance Corporate News