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NEWS

IntraBio’s Aqneursa Receives the CHMP’s Positive Opinion for Treating Niemann-Pick Disease Type C (NPC)

Shots:The CHMP has recommended Aqneursa (levacetylleucine) for treating adults & pediatric pts with NPC; additional global submissions are planned for 2025 & beyondApproval 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.,…
July 30, 2025

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NEWS

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 …
September 25, 2024

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