Shionogi expands rare disease portfolio with IntraBio buyout
Shionogi has announced plans to acquire IntraBio, a biopharmaceutical company developing therapies for neurodegenerative diseases, for $2 billion.
The transaction will add Aqneursa (levacetylleucine) and IntraBio’s capabilities in the rare disease area to Shionogi’s growing portfolio.
Aqneursa was approved by the FDA in September 2024 for neurological manifestations of Niemann-Pick disease Type C (NPC) in adults and paediatric patients and by the European Medicines Agency (EMA) in January 2026.
It was approved by the FDA for the treatment of Ataxia in adults and paediatric patients diagnosed with Ataxia-Telangiectasia (A-T) in September 2026. The drug is currently under review by the EMA for this indication.
“The planned acquisition of IntraBio actively demonstrates Shionogi’s solid commitment to building a leading global rare disease business,” said Isao Teshirogi, CEO of Shionogi. “Shionogi has a strong record of delivering innovative medicines for challenging infectious diseases, and we are applying that same focus and determination to rare diseases. Bringing Aqneursa to Shionogi after our acquisition of Radicava will deepen our commitment to rare disease communities, expand our capabilities and strengthen our portfolio as we advance future innovation for patients with significant unmet needs.”
IntraBio also has clinical programmes in Pompe disease, Fragile X syndrome, and Jordan’s syndrome, as well as additional early-stage rare neurodegenerative disease programmes recently added to the pipeline.
“This milestone gives us the opportunity to build on the meaningful foundation IntraBio has established with the Niemann-Pick Type C community and to deliver the first and only therapy for Ataxia-Telangiectasia to a community that has lived for many years without options to treat the diverse and debilitating symptoms caused by this disorder,” said Nathan McCutcheon, President and CEO, Shionogi Inc. “In addition to Aqneursa, the capabilities and resources gained through this acquisition would help to accelerate the development of new treatment options.”