# The FDA approved a new medicine from Denali Therapeutics for a condition called Hunter syndrome.

*biotech · news · 2026-03-25 · STAT*

## Key points

- The FDA approved Denali Therapeutics' Avlayah for Hunter syndrome despite recent tough standards.
- Denali's Avlayah approval contrasts with Regenxbio's gene therapy rejection for the same condition.
- The FDA cited insufficient clinical data when rejecting Regenxbio's Hunter syndrome gene therapy.
- Advocates feared Avlayah would be rejected due to a trend of increased FDA scrutiny.

The Food and Drug Administration on Wednesday approved a new medicine from Denali Therapeutics for a condition called Hunter syndrome, a notable decision by the agency as it has recently taken tougher stances on rare disease drugs. Last month, the FDA rejected a Hunter syndrome gene therapy from Regenxbio, saying that the company needed to produce more clinical data, a higher standard of evidence that could take the company years to meet. Advertisement The gene therapy’s denial — on top of other FDA rejections of rare disease therapies and signals that officials were scrutinizing the drugs more closely — left advocates worried about the fate of Denali’s drug, called Avlayah.

**Companies:** Denali Therapeutics, Regenxbio
**Countries:** United States

[Read the full story on STAT](https://www.statnews.com/2026/03/25/denali-therapeutics-hunter-syndrome-rare-disease/)

---

Canonical: https://newsio.io/n/a6ac1e36-f428-4f13-8484-15173ce28bb7/the-fda-approved-a-new-medicine-from-denali-therapeutics-for-a-condition-called
Summarized by Newsio from STAT. https://newsio.io/how-it-works