Shionogi to acquire IntraBio for US$2B to expand rare disease portfolio
IntraBio will become a wholly owned subsidiary of Shionogi Inc., the Japanese group’s US-based subsidiary in New Jersey.
USA—Japanese pharmaceutical company Shionogi plans to acquire IntraBio for US$2 billion to expand its rare disease business and gain global rights to neurological drug Aqneursa.
The proposed transaction would give Shionogi ownership of all outstanding shares of IntraBio, along with worldwide intellectual property and commercialisation rights for Aqneursa (levacetylleucine).
IntraBio will become a wholly owned subsidiary of Shionogi Inc., the Japanese group’s US-based subsidiary in New Jersey.
Aqneursa is a small molecule medicine developed to address metabolic dysfunction in the central nervous system (CNS).
It treats neurological manifestations of Niemann-Pick disease type C (NPC), a rare genetic disorder in which harmful lipids accumulate in cells, progressively damaging the nervous system and other organs.
Expanding neurological disease treatments
The US Food and Drug Administration (FDA) first approved Aqneursa for NPC in 2024.
The medicine has since received approval in the European Union for the same indication and US approval for ataxia-telangiectasia (A-T), another rare genetic disorder that affects movement, coordination, and immune function.
European regulators are reviewing an application to approve Aqneursa for A-T.
The acquisition would expand Shionogi’s presence in rare diseases, a market in which the company has recently increased its investment.
Earlier in 2026, the Japanese drugmaker agreed to acquire Tanabe Pharma’s amyotrophic lateral sclerosis (ALS) medicine, Radicava (edaravone), in a US$ 2.5 billion transaction.
Adding IntraBio would broaden its neurological disease portfolio and give it access to treatments targeting conditions with limited therapeutic options.
The deal would also give Shionogi access to IntraBio’s rare disease development expertise, potentially supporting its efforts to advance medicines for conditions with significant unmet medical needs.
Its pipeline includes programmes targeting Fragile X syndrome, Pompe disease and Jordan’s syndrome, alongside early-stage research into neurodegenerative diseases.
Commercial reach and pipeline development
Shionogi intends to use its international commercial infrastructure to support Aqneursa’s reach across the US, Europe and other markets.
Acquiring the drug’s global rights would give the company control over its commercial development and future expansion into additional indications, subject to regulatory approvals.
The transaction also reflects the pharmaceutical industry’s interest in acquiring medicines that have already secured regulatory approval, particularly in specialist markets where patient populations are small and treatment options remain limited.
Such acquisitions can provide established products and development opportunities while allowing buyers to build expertise in complex disease areas.
Nathan McCutcheon, president and chief executive officer of Shionogi Inc., said the acquisition would build on the foundation IntraBio had established with the NPC community and support the company’s expansion into rare diseases.
He also highlighted the opportunity to bring treatment options to people living with A-T.
Beyond Aqneursa, McCutcheon said the deal’s capabilities and resources could accelerate the development of additional treatments.
“With this transaction, we deepen our commitment to serving rare disease communities today and delivering innovation tomorrow,” he said.
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