Daily Briefing

Around the nation: New gene therapy slows progression of Huntington's disease


A new experimental treatment from gene therapy company uniQure was able to slow the progression of Huntington's disease for the first time, in today's bite-sized hospital and health industry news from Georgia, Maryland, and Massachusetts. 

  • Georgia: CDC has made a strong recommendation for lenacapavir, a new long-acting pre-exposure prophylaxis (PrEP), based on a high certainty of evidence. "[Lenacapavir] is a highly effective and safe PrEP option administered every 6 months, which provides the potential to improve PrEP adherence and thus enhance HIV prevention in the United States," said CDC's PrEP Guidelines Work Group. Lencapavir has been hailed as a game changer in the HIV space, with Mitchell Warren, executive director of the HIV advocacy nonprofit AVAC, saying that it is "the single best opportunity in 44 years of HIV prevention." However, despite strong evidence of the drug's benefits, as well as CDC's recommendations, it's not clear where health insurers will cover the drug, which costs $28,000 a year. Although some private pharmacy benefit managers (PBMs) have added lenacapavir to their formularies, CVS Caremark, one of the largest PBMs in the country, declined to include it on the company's list of covered PrEP therapies. CVS Health also announced that it would not include lenacapavir in its commercial or Affordable Care Act plans. In response to CVS' decision, several advocacy organizations sent a letter to the company, calling for it to rethink its decision. "Ensuring comprehensive, zero-cost PrEP access is both a legal requirement and a critical public health imperative," the organizations said. (Rudd, MedPage Today, 9/24)
  • Maryland: According to CMS, enrollment in Medicare Advantage (MA) plans is expected to decrease next year, marking the first decline in years. Currently, insurers estimate 34 million people will sign up for MA plans in 2026, declining from 34.9 million in 2025. The number of available MA plans will also slightly fall from 5,633 in 2025 to roughly 5,600 in 2026. According to analysts from TD Cowen, next year could be the start of declining enrollment in MA plans, with enrollment predicted to decrease by 2.6% year over year. At the same time, the average monthly premium for MA plans is expected to decrease in 2026, going from $16.40 to $14. Although CMS said it expects premiums, benefits, and plan choices to "remain stable" next year, analysts predict that there will be benefit reductions when CMS releases its plan finder tool. Open enrollment for Medicare plans will run from Oct. 15 to Dec. 7. (Olsen, Healthcare Dive, 9/29)
  • Massachusetts: Gene therapy company uniQure last week announced that its experimental treatment AMT-130 was able to slow the progression of Huntington's disease in a clinical trial — making it the first treatment to do so. Huntington's disease is a genetic disorder that often develops when people are in their 40s, with symptoms including jerky and involuntary movements, behavior disruptions, and cognitive decline. To date, a few drugs have been approved for Huntington's disease, but they largely focus on managing symptoms rather than slowing progression. In a clinical trial, 17 patients were given a high dose of AMT-130 through brain surgery. The treatment is a harmless virus that delivers microRNA into brain cells to stop production of the protein by the Huntington's gene. Overall, AMT-130 was able to slow the progression of the disease by 75%. "The key thing I think — the 75 percent slowing means that people will be able to stay in work longer, they'll be able to function longer, they'll be able to maintain their independence," said Sarah Tabrizi, a scientific advisor to uniQure and director of the University College London Huntington's Disease Center. "We want to be able to treat people, eventually, at Stage 0 and 1, decades before they show any signs and symptoms with such therapy. So we may be able to prevent the symptoms from ever occurring." Although the findings are only early results from a small trial, the company said it plans to submit data seeking approval for the treatment from U.S. regulators in the first quarter of next year. (Johnson, Washington Post, 9/24)

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