Dive Brief:

• Shionogi is expanding in rare diseases, announcing Monday a deal to acquire Texas-based IntraBio for a drug called Aqneursa that has gained approvals in two inherited disorders.
• Per deal terms, Shionogi will pay IntraBio’s shareholders $2 billion in cash and turn the company into a wholly owned subsidiary of its New Jersey-headquartered U.S. division. Shionogi said it will review the transaction’s effects on its financial outlook for the current fiscal year, which ends next March.
• The transaction will help expand a rare disease business Shionogi launched with the acquisition last year of the ALS drug Radicava from Tanabe Pharma. Aside from Radicava, Shionogi’s U.S. portfolio includes the COVID-19 medicine Xocova and a treatment for complicated urinary tract and hospital acquired respiratory infections.

Dive Insight:

Shionogi sees rare disease treatments playing a larger role in its future.

The addition of Aqneursa bolsters the company’s commercial-stage portfolio. An experimental treatment acquired through a 2020 buyout of Tetra Therapeutics is currently in a Phase 3 trial for the neurological condition Fragile X syndrome. Shionogi, in its statement, also touted “additional early-stage rare neurodegenerative disease programs recently added to the pipeline.”

“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,” CEO Isao Teshirogi said in a statement.

Aqneursa emerged from research at the University of Oxford. The drug combats the toxic buildup of lipids in the cells of people with Niemann-Pick disease type C, a condition that affects about 1 per million people in the U.S.

In a clinical trial, use of Aqneursa was associated with improved patient scores on an evaluation of movement disorders in people with the disease, which provided the data necessary to win a U.S. approval in 2024.

The company was later able to show that Aqneursa had a similar benefit in people with a more common disorder known as Ataxia-Telangiectasia, spurring a clearance in that indication in September.

IntraBio reported sales of $68 million in 2025, Shionogi said.