IntraBio’s Aqneursa Receives the US FDA’s Approval for Treating Niemann-Pick Disease Type C
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
- 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
Related News:- Zevra Therapeutics Resubmits Arimoclomol’s NDA to the US FDA for Treating Niemann-Pick disease Type C (NPC)
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
