Shots:
Shionogi announced a planned acquisition of IntraBio for $2.0B, adding AQNEURSA (levacetylleucine) and IntraBio’s rare disease capabilities to its portfolio; the transaction is expected to close between November and December 2026, subject to customary conditions and regulatory approvals
AQNEURSA is approved in the US and EU for neurological manifestations of Niemann-Pick disease Type C…
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…
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.,…

