Aqneursa (levacetylleucine) for Ataxia-telangiectasia
The FDA is expected to decide on Aqneursa for Ataxia-telangiectasia by September 19, 2026, under a sNDA from IntraBio.
What is being decided
IntraBio has a sNDA under FDA review for Aqneursa (levacetylleucine) in Ataxia-telangiectasia. Already approved for Niemann-Pick disease type C; this expansion would cover ataxia-telangiectasia under priority review.
What each outcome would mean
An approval starts a second race rather than ending the first one: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. Our guide to the 90 days after a rare disease approval explains the timeline and the moves families can make on day 1.
A complete response letter would mean the FDA declined to approve in the application's current form. CRLs are often about manufacturing or data presentation rather than efficacy, and resubmission is common. Either way, this page updates with the outcome and what it means.
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Frequently asked questions
When will the FDA decide on Aqneursa?
The FDA's target decision date (PDUFA date) for Aqneursa in Ataxia-telangiectasia is September 19, 2026, disclosed by IntraBio. The agency can act before this date and occasionally runs past it.
What is Aqneursa being reviewed for?
IntraBio submitted a sNDA for Aqneursa in Ataxia-telangiectasia. Already approved for Niemann-Pick disease type C; this expansion would cover ataxia-telangiectasia under priority review.
What happens after the Aqneursa decision?
If approved, availability is not immediate: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. If the FDA issues a complete response letter, the application was not approved in its current form; CRLs are often about manufacturing or data presentation rather than efficacy, and sponsors frequently resubmit. This page updates with the outcome either way.
Where this date comes from
The FDA does not publish PDUFA dates; companies disclose them. This one comes from IntraBio announcement. Dates can move, and the FDA can act early or late. This page rechecks against our calendar, which is re-verified weekly.