Shionogi agrees to buy IntraBio for $2 billion to own Aqneursa, the only FDA-approved A-T ataxia drug
Shionogi agreed to pay $2 billion for IntraBio, maker of Aqneursa, the first FDA-approved drug for ataxia in A-T. The April Radicava deal came with a sales forecast. This one comes with no disclosed sales figure to test the price against.
The Scientist · Science desk
What happened
- Aqneursa, the brand name for levacetylleucine, won FDA approval in September 2024 for neurological manifestations of Niemann-Pick disease type C, then EU approval in January.
- The European Medicines Agency is now reviewing Aqneursa for adults and children with A-T, after the FDA's September 18 label expansion.
- Two months before the deal, Shionogi chief executive Isao Teshirogi told Bloomberg News the company was actively pursuing at least three acquisition opportunities.
- Shionogi shares fell about 4.5% in Tokyo on Monday, to 2,661 yen, then gained 3.8% on Tuesday to reach 2,763 yen.
- Canaccord Genuity analyst Sumant Kulkarni wrote that companies with approved products for rare indications hold "significant scarcity value" for strategic buyers and investors.
Why it matters
- What it limits constraint Shareholders can check the Radicava price at roughly 3.6 times expected sales, but they cannot run the same check on the $2 billion until Shionogi discloses what Aqneursa earns.
- Who's exposed exposure Kulkarni ties the sale's timing to the A-T approval, so the pending EMA decision on A-T bears directly on how much of what Shionogi bought applies in Europe.
- What it sets up precedent If approved rare-disease assets carry the scarcity premium Kulkarni describes, other independent holders of such approvals become candidates for Shionogi's remaining acquisition search.
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The closest comparison for the IntraBio price is the deal Shionogi signed in April. It agreed to pay Tanabe Pharma $2.5 billion, plus a potential royalty, for the ALS drug Radicava [6]. Shionogi said that drug would add about $700 million in annual global sales in the fiscal year that began April 1 [12]. That is roughly 3.6 times a year of sales [17]. Kulkarni, the Canaccord Genuity analyst, wrote that he was "encouraged by the upfront consideration" for IntraBio [14]. The report does not split the $2 billion into upfront and contingent payments, and it does not include Aqneursa's sales, so the same multiple cannot be computed.
The 59% figure attached to Radicava compares two different measures. GEN reported that the $700 million would be a 59% leap from Shionogi's entire fiscal 2025 EBITDA of $1.187 billion [7]. That divides one drug's revenue by the whole company's earnings. Radicava adds to EBITDA only what is left after the cost of making and selling it, so its real addition to earnings is smaller than 59%. The forecast also assumes growth. Radicava brought in $597.6 million in fiscal 2025 [13], and $700 million would be about 17% more [19].
Aqneursa's value to Shionogi depends on its label. One molecule, levacetylleucine, now holds US approvals for two rare neurological diseases, and both are limited to patients weighing at least 15 kg [4][2]. An approval means a regulator judged the benefit worth the risk in the people who were studied. It does not tell you how large the benefit was, or how many patients will be prescribed the drug. Kulkarni linked the deal's timing to the newest indication. "In fact, we were surprised that IntraBio had remained independent for the time it did, but the timing makes sense given the recent approval for A-T, which adds another leg to its story," he wrote [10].
Teshirogi presented the purchase as strategy. "The planned acquisition of IntraBio actively demonstrates Shionogi's solid commitment to building a leading global rare disease business," he said in a statement [5]. He had already told Bloomberg News that money was not what limited him. "From a cash flow perspective, we've become a company with plenty of financial firepower," he said [15]. Counting Radicava, Shionogi has committed $4.5 billion since April to two marketed drugs for rare neurological diseases [16]. The share price fell on Monday and rebounded on Tuesday, ending 23 yen, about 0.8%, below where it started on Monday [18].
What to watch
- The EMA decision on Aqneursa for A-T; approval would give the drug the same two indications in Europe that it holds in the US.
- Any Shionogi disclosure of Aqneursa revenue, or of how the $2 billion splits between upfront and contingent payments.
- Whether another of the three or more acquisition opportunities Teshirogi described becomes a signed deal.
Clarity's read
What the record supports and how the coverage leans. The claims behind it follow.
Reality
- Evidence70
- Adoption35
- Hype gap+20
- Incentives55
- Confidence65
Claim ledger
Ranked by verification strength, evidence, and original report placement.
- [1]
Shionogi has agreed to acquire IntraBio for $2 billion, to expand its rare disease portfolio with a drug marketed in the US and EU for neurological manifestations of Niemann-Pick disease type C.
- [2]
On September 18 the FDA approved a supplemental New Drug Application expanding Aqneursa's label, making it the first and to date only drug approved for the treatment of ataxia in ataxia-telangiectasia (A-T) patients weighing 15 kg or more.
- [3]
Aqneursa is now under review by the EMA for adults and children diagnosed with A-T.
- [4]
Aqneursa (levacetylleucine) was approved by the FDA in September 2024 for neurological manifestations of Niemann-Pick disease type C in adults and children weighing 15 kg or more, and by the EMA in January.
- [5]
"The planned acquisition of IntraBio actively demonstrates Shionogi's solid commitment to building a leading global rare disease business," Isao Teshirogi, Shionogi's president, CEO and representative director, said in a statement.
- [6]
In April Shionogi agreed to acquire Radicava (edaravone), an ALS treatment, from Tanabe Pharma for $2.5 billion and a potential royalty on future sales.
- [7]
GEN wrote that the roughly $700 million would represent a 59% leap from Shionogi's entire FY2025 EBITDA of 187.72 billion yen ($1.187 billion).
- [8]
Shionogi shares on the Tokyo Stock Exchange fell about 4.5% Monday, from 2,786 yen to 2,661 yen, then rose 3.8% Tuesday to 2,763 yen.
- [9]
"In summary, this transaction underscores our view that companies with approved products for rare indications, neuro or otherwise, present significant scarcity value for strategics and investors alike," Sumant Kulkarni of Canaccord Genuity wrote in a research note.
- [10]
"In fact, we were surprised that IntraBio had remained independent for the time it did, but the timing makes sense given the recent approval for A-T, which adds another leg to its story," Kulkarni wrote.
- [11]
Two months before the IntraBio deal, Teshirogi told Bloomberg News that Shionogi was "actively pursuing" at least three acquisition opportunities.
- [12]
Shionogi said the Radicava deal would add approximately $700 million in annual global sales during its current 2026 fiscal year, which began on April 1.
- [13]
Radicava generated 94.491 billion yen ($597.582 million) in the 2025 fiscal year that ended March 31.
- [14]
"We are not entirely surprised by this development, but are encouraged by the upfront consideration," Kulkarni wrote.
- [15]
"From a cash flow perspective, we've become a company with plenty of financial firepower. If a good opportunity comes along, there's absolutely no reason for us to hesitate over any M&A," Teshirogi told Bloomberg News.
- [16]
Shionogi has committed about $4.5 billion since April to the Radicava and IntraBio deals combined.
- [17]
Shionogi paid roughly 3.6 times expected annual sales for Radicava.
- [18]
After Tuesday's rebound, Shionogi shares stood 23 yen, about 0.8%, below Monday's starting price.
- [19]
The $700 million Radicava sales forecast is about 17% above its fiscal 2025 revenue.
Sources
2 independent publishers whose own reporting we read for this story.
- genengnews.comShionogi Grows Rare Disease Portfolio with $2B IntraBio Acquisition
1 article · October 6, 2026
- qz.comShionogi is buying rare disease biotech IntraBio for $2 billion
1 article · October 5, 2026
Topics and entities
Topics
- Ataxia-telangiectasia
- Niemann-Pick disease type C
- Rare disease drug M&A