Shots:
- The EC has approved Aqneursa (levacetylleucine), available as 1g granules for oral suspension, with or without miglustat for treating adults & pediatric pts (≥6yrs.; ≥20kg) with NPC
- Approval was backed by P-III (IB1001-301) crossover study assessing Aqneursa vs PBO in NPC pts, which showed improved neurological symptoms & function after 12wks. as measured by SARA, with the ongoing OLE phase showing sustained disease-modifying & neuroprotective effects
- Also, observational analyses vs a natural history cohort showed Aqneursa reduced annual disease progression by 118% at 1yr. measured by the 5-domain NPC-CSS, with a comparable sustained effect observed after 2yrs.
Ref: Businesswire | 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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