Six advocacy groups have asked the federal government to sidestep a handful of patents on a pricey drug used to treat a rare form of muscular dystrophy as part of an ongoing campaign to provide wider access to high-cost medicines.

In a letter sent on Wednesday to the Department of Health and Human Services, the groups argued that the patents — which are either owned or licensed by Sarepta Therapeutics (SRPT) — failed to disclose federal funding for grants that were used to develop a drug known as Exondys 51 for treating Duchenne muscular dystrophy. This is an accompanying memo sent to HHS.

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  • As a matter of healthcare ethics and public policy, the government should note that Sarepta, in its third quarter investor call, projected that they would generate $300M in revenues from Exondys 51 in 2018. The patient population or Exondys 51 is only 13% of the Duchenne population of only 2,000 – 3,000 people, or perhaps fewer than 300 people. Especially since Sarepta’s drug was developed from NIH funded research, this unconscionable pricing must be stopped.

  • President Trump, please make this very critical medicine affordable for anyone to purchase. Thank you.

  • In light of the pricing in this post, the fears reported earlier in the day of a tariff on Chinese raw materials causing rise in drug prices seem ….. overblown at a minimum and ludicrous as well.

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