# FDA 批准了 Denali Therapeutics 用於 Hunter 綜合症的新藥。

*biotech · news · 2026-03-25 · STAT*

## Key points

- 儘管近期標準趨嚴，FDA 仍批准 Denali Therapeutics 的 Avlayah 用於 Hunter 綜合症。
- Denali 的 Avlayah 獲批與 Regenxbio 同病症基因療法被拒形成對比。
- FDA 拒絕 Regenxbio 的 Hunter 綜合症基因療法，理由是臨床數據不足。
- 倡議者擔心 Avlayah 會因 FDA 審查趨嚴而被拒絕。

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/)

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