Shionogi to Acquire IntraBio, Adding Aqneursa to Rare Disease Portfolio

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Shionogi will acquire IntraBio, the company said on Oct. 5, making IntraBio a wholly owned Shionogi subsidiary.

What Shionogi Will Add

Most prominently, the pending transaction adds Aqneursa (levacetylleucine) to Shionogi’s expanding rare disease portfolio. Aqneursa just gained U.S. FDA approval in September for treatment of ataxia in adult and pediatric patients with ataxia-telangiectasia (A-T).

A-T is a rare disease affecting an estimated 1 in 40,000 people. It is a progressive, prematurely fatal autosomal-recessive neurodegenerative disorder. As such, it fits IntraBio’s profile of developing and commercializing therapies for neurodegenerative diseases.

Aqneursa Just One Part of Portfolio

Aqneursa’s first FDA approval came in September 2024, for neurological manifestations of Niemann-Pick disease Type C (NPC). NPC is a slowly progressive lysosomal disorder for which principal manifestations are age dependent.

Shionogi CEO Isao Teshirogi, PhD, said the planned acquisition of IntraBio will help Shionogi build a global rare disease business.

Teshirogi put this deal in context with another, the acquisition of Radicava (edaravone) from Tanabe Pharma in April. Radicava is a treatment approved by the FDA and other global regulatory authorities, for amyotrophic lateral sclerosis (ALS).

“Shionogi has a strong record of delivering innovative medicines for challenging infectious diseases,” Teshirogi said. “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. [It will] expand our capabilities and strengthen our portfolio as we advance future innovation for patients with significant unmet needs.”

IntraBio Brings Rare Disease Knowledge

The IntraBio acquisition also brings its more general rare disease capabilities—outside of Aqneursa specifically—to Shionogi. However, as for Aqneursa, the European Medicines Agency currently is reviewing the treatment, for adult and pediatric patients with A-T.