FDA delays decision on troriluzole to Q4

Troriluzole could become the first FDA-approved treatment for spinocerebellar ataxia (SCA)

Biohaven Ltd. announced that the U.S. Food and Drug Administration’s (FDA) Division of Neurology 1 has extended the Prescription Drug User Fee Act (PDUFA) date for the troriluzole New Drug Application (NDA) by 3 months to allow time for a full review of recent submissions. The FDA is planning an advisory committee meeting to discuss the application, with a decision now expected in Q4 2025. No new concerns were raised.

Troriluzole has previously received Fast Track, Orphan Drug Designation, and Priority Review. Clinical data presented in the NDA showed a highly favorable benefit–risk profile, with the once-daily oral pill slowing disease progression by 50–70% (f-SARA scale) and reducing fall risk.

Vlad Coric, Chairman and CEO of Biohaven, stated, “SCA is a devastating neurodegenerative disease that has affected generations of families and has no current approved therapy. We are committed to bringing the first treatment to patients and families affected by SCA... We are especially grateful to the patients, families, and leading neurologists who participated in our studies over an 8-year period and the broader SCA community and patient advocacy groups, including the National Ataxia Foundation.”

Why did the FDA extend the PDUFA date for troriluzole?

The FDA extended the PDUFA date by 3 months to allow time for a full review of Biohaven’s recent submissions related to information requests.

What clinical benefits has troriluzole shown in trials?

Troriluzole slowed disease progression by 50–70% as measured by the f-SARA scale and reduced the risk of falls.

When is the FDA decision on the troriluzole NDA now expected?

The FDA decision is now expected in Q4 2025.

What designations has troriluzole received from the FDA?

Troriluzole has received Fast Track, Orphan Drug Designation, and Priority Review.