Shots:
- The US FDA has approved Aqneursa (levacetylleucine) for the treatment of ataxia in adult & pediatric pts (≥15kg) with ataxia-telangiectasia (A-T)
- Approval was backed by the P-III (IB1001-303) trial assessing Aqneursa vs PBO in 73 pts (4-50yrs.) with genetically confirmed A-T, which met its 1 & key 2EPs & showed that Aqneursa has a favorable safety profile
- Trial showed improved signs & symptoms of ataxia, with functional benefits evident within 12wks.; SARA scores were improved (treatment difference (TD): −1.9), while FDA-assessed fSARA also improved (TD: −0.6), with consistent benefits across 2EPs & subgroups
Ref: Businesswire | Image: IntraBio | Press Release
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