Bernie Sanders Calls out This Drug Company Over Its 'Orphan Drug'

A drug company is being accused of exploiting a federal program meant to encourage research into rare diseases came, and on Monday two U.S. lawmakers started demanding answers.


Sen. Bernie Sanders (I-Vt.) and Rep. Elijah Cummings (D-Md.) allege that Marathon Pharmaceuticals — which sells deflazacort, a drug for the genetic muscle deterioration disorder known as Duchenne — 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."

The orphan drug program is not meant to "provide companies like Marathon with lucrative market exclusivity rights for drugs that have been available for decades," Sanders said in a press release.

What you need to know about the government's orphan drug program.

  • In 1983, President Ronald Reagan signed the Orphan Drug Act in an effort to bolster the development of drugs that treat rare conditions affecting populations of 200,000 or fewer — a market that had been largely ignored due to a lack of financial incentive.
  • To make orphan drug development more appealing to drug companies, the government offered subsides amounting to $2 million per drug, tax credits, fee wavers, and seven-year monopolies, meaning no other companies could sell the drug for that period.
  • But some companies have exploited the orphan drug program, according to a 2016 investigative report by Kaiser Health News that found certain companies claim only one medical use of a drug to the Food and Drug Administration (FDA) — one that qualifies it for an "orphan drug" status — but then claim another to sell to a mass market after the exclusivity period ends.

"The industry has been gaming the system by slicing and dicing indications so that drugs qualify for lucrative orphan status benefits," Dr. Martin Makary wrote in a commentary for the American Journal of Clinical Oncology. "As a result, funding support intended for rare disease medicine is diverted to fund the development of blockbuster drugs."

In the case of Marathon's deflazacort, the FDA's orphan drug database lists three claimed uses for the drug. The FDA approved the drug as an orphan drug for different conditions in 2010, 2013, and 2015.

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The drug's list price stands at $89,000 per year. The company argues that this is "modestly priced for an orphan drug," according to Sanders' press release. But the senator points out that the drug is available for as little as $1,000 per year in other countries.

In a statement emailed to ATTN:, Marathon announced that it was “pausing our commercialization efforts in order to meet with Duchenne community leaders and explain our commercialization plans, review their concerns, discuss all options, and move forward with commercialization based on the resulting plan of action."

The company also emphasized that it expects patients to pay no more than $20 for a prescription after insurance and that any profits from the drug will be reinvested in further research in Duchenne.