Shots:
- The US FDA has granted approval to Aqneursa (levacetylleucine) for treating adults & pediatric patients (≥15kg) with neurological manifestations of Niemann-Pick disease type C (NPC)
- Approval was supported by pivotal P-III (IB1001-301) study assessing the effect of Aqneursa vs PBO on neurological symptoms & functioning in pediatric (≥4yrs.) & adults (n=60) with confirmed NPC
- The study reached all 1 & 2EPs, depicting improved neurological symptoms & functional benefits at 12wks. as well as greater improvement in fSARA scores, with a mean difference of -0.4. Results were published in the New England Journal of Medicine
Ref: IntraBio | Image: IntraBio| Press Release
Related News:- Zevra Therapeutics Receives the US FDA’s Approval of Miplyffa (Arimoclomol Citrate) for Treating Niemann-Pick Disease Type C (NPC)
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