Regulatory
FDA Accepts IntraBio's sNDA for Levacetylleucine (AQNEURSA) for Ataxia-Telangiectasia with Priority Review
September 18, 2026
Biotech
Regulatory
Key Facts
Signal Type
Regulatory
Industry
Biotech
Companies
IntraBio
Date
September 18, 2026
The FDA has accepted IntraBio's supplemental new drug application (sNDA) for levacetylleucine (AQNEURSA) as a treatment for ataxia-telangiectasia (A-T) in adult and pediatric patients. The agency granted Priority Review, setting a PDUFA target action date of September 19, 2026.
The application is supported by data from the pivotal phase 3 IB1001-303 trial, which met its primary and key secondary endpoints, showing statistically significant improvements in neurologic function and patient-reported outcomes.
IntraBio, a US-based biotech operating company in Austin, Texas, is the sponsor. The therapy targets A-T, a rare inherited neurodegenerative disorder affecting both children and adults.
If approved, AQNEURSA would be the first FDA-approved therapy specifically indicated for A-T, addressing a significant unmet medical need for thousands of patients and families.
This regulatory milestone positions IntraBio for a potential commercial launch in late 2026, creating immediate opportunities for service providers.
Monitor the PDUFA date of September 19, 2026, for a final approval decision. Also watch for the release of full peer-reviewed efficacy and safety data from the phase 3 trial, which could influence payer and prescriber adoption.
Long-term safety outcomes from the extension study will be critical, as will any competitive developments in the A-T treatment landscape.
Source:
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