Shots:
- The CHMP has recommended Aqneursa (levacetylleucine) for treating adults & pediatric pts with NPC; additional global submissions are planned for 2025 & beyond
- Approval was backed by P-III (IB1001-301) study assessing Aqneursa vs PBO in pts (≥4yrs.; n=60) with NPC, which showed improved neurological symptoms & function across all 1 & 2EPs within 12wks., with long-term extension phase showing disease-modifying & neuroprotective effects. IB1001-301 trial data was published in The NEJM
- Also, Aqneursa is being evaluated in a P-III trial for Ataxia-Telangiectasia, which completed recruitment in under 2mos., over-enrolled by 167%, & is expected to report data in Q1’26
Ref: BussinessWire| Image: IntraBio| Press Release
Related News:- IntraBio’s Aqneursa Receives the US FDA’s Approval for Treating Niemann-Pick Disease Type C
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