FDA Under New Leadership Revives Huntington's Gene Therapy, Moderna mRNA Flu Vaccine

The FDA under acting Commissioner Kyle Diamantas is reversing course on two high-profile drug reviews that had stalled under the previous leadership of Marty Makary and Vinay Prasad. The most significant development: UniQure announced the agency will now consider accelerated approval for its Huntington's disease gene therapy — a treatment that affects roughly 40,000 Americans and for which there are currently zero therapies that slow disease progression.

The gene therapy had shown striking results in a three-year study, slowing disease progression by 75% compared to an external control group. Despite that data, Prasad demanded a randomized controlled trial requiring a placebo group to undergo sham brain surgery — a design widely considered unethical. When Huntington's patients publicly objected, an FDA spokesperson attacked them on social media as 'the swamp,' and a senior official anonymously accused UniQure of presenting manipulated data for a therapy he called a failure. Makary went further, claiming on television the treatment showed 'no benefit.'

The hostility toward UniQure became emblematic of a broader pattern: arbitrary rejections, shifting approval goalposts, and open antagonism toward patients and industry that was chilling biotech investment at a moment when the U.S. faces mounting competition from China in life sciences. That pattern ultimately contributed to the White House ousting both Makary and Prasad this spring.

Diamantas has moved quickly to signal a reset. He met with rare disease advocacy groups this month and convened an outside advisory committee to review Moderna's mRNA flu vaccine — a step Prasad had blocked by overruling career FDA staff and refusing to even review the application. The advisory panel voted unanimously to recommend approval. The mRNA vaccine was 27% more effective than a standard flu shot in a large clinical trial, produces a more durable immune response, and could allow vaccines to better match circulating strains. If the FDA grants approval promptly, the shots could be available as early as this fall.