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NEWS

Shionogi to Acquire IntraBio for $2B to Expand Rare Disease Portfolio

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…
11 hours ago

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INSIGHTS+

EMA Marketing Authorization of New Drugs in January 2026    

Shots:  The European Commission approved multiple therapies across vaccines, rare diseases, endocrinology, and immunology, including products from BioNet, Sanofi, Fondazione Telethon, IntraBio, and Ionis / Otsuka.  The Committee for Medicinal Products for Human Use also issued positive opinions for late-stage assets from UCB and Gedeon Richter, with additional recommendations supporting Sanofi’s Rezurock.  Decisions were backed by strong Phase II/III data…
February 17, 2026

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NEWS

IntraBio Reports Topline P-III (IB1001-303) Trial Data on Aqneursa for Ataxia-Telangiectasia

Shots: IntraBio has reported the topline P-III (IB1001-303) trial data on Aqneursa (levacetylleucine) vs PBO in pediatric & adult pts with Ataxia-Telangiectasia (A-T) Trial met the 1EP with a significant –1.88 SARA improvement vs PBO (–1.92 vs –0.14) after 12wks.& achieved 2EPs, showing meaningful gains on ICARS (–4.22 vs –1.69) & CGI-I (–0.6 vs –0.2)…
January 22, 2026

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NEWS

IntraBio’s Aqneursa Receives the EC Approval for Treating Niemann-Pick Disease Type C (NPC)

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…
January 22, 2026

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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 & 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.,…
July 30, 2025

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IntraBio
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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