Edaravone

Shionogi’s potential acquisition of IntraBio follows on from its purchase of Tanabe Pharma’s ALS med, Radicava. Credit: agustin.photo / Shutterstock.com. Japanese pharma company, Shionogi, is set to acquire IntraBio in a multibillion-dollar deal, adding neurological and movement disorder drug, Aqneursa (levacetylleucine), to its portfolio and marking its fortification in the rare disease space. As per the transaction, Shionogi will pay $2bn to acquire all of IntraBio’s outstanding shares, which will see the company secure the worldwide IP and commercialisation rights to Aqneursa – a small molecule drug that acts by addressing metabolic dysfunction in the central nervous system (CNS). IntraBio will become a wholly owned subsidiary of Shionogi Inc., a New Jersey-based subsidiary of Shionogi. Aqneursa was first approved by US regulators to treat neurological manifestations of a rare lysosomal disorder called Niemann Pick disease type C (NPC), which is characterised by the gradual buildup of harmful lipids in the cells, in 2024. Since then, the drug has also secured the EU regulatory greenlight in NPC, as well as the US Food and Drug Administration (FDA) go-ahead in rare genetic movement disorder, ataxia-telangiectasia (A-T). EU regulators are currently reviewing an approval application for Aqneursa in A-T. By adding Aqneursa to its portfolio of medicines, Shionogi would double down on its new-found intentions to break into the rare disease space. This build on its $2.5bn acquisition of Tanabe Pharma’s amyotrophic lateral sclerosis (ALS) medication, Radicava (edaravone) , which came earlier this year. The Japanese drugmaker also hopes to leverage IntraBio’s expertise in the rare disease space to fortify its current pipeline, which includes drugs for conditions with high unmet needs such as Fragile X syndrome, Pompe disease and Jordan’s syndrome, alongside its early-stage programmes for neurodegenerative diseases. According to Nathan McCutcheon, President and CEO of Shionogi Inc, bringing Aqneursa to the company would help it to continue its expansion into the rare disease market across Europe, the US and further afield, building on the “meaningful foundation IntraBio has established with the NPC community,” while delivering the first and only therapy for A-T. “In addition to Aqneursa, the capabilities and resources gained through this acquisition would help to accelerate the development of new treatment options. With this transaction, we deepen our commitment to serving rare disease communities today and delivering innovation tomorrow,” McCutcheon concluded.

iStock, Cristina Gaidau With its acquisition of IntraBio, Shionogi will gain ownership over Aqneursa, which could hit peak global sales of $1 billion across its two indications in Niemann-Pick disease type C and ataxia-telangiectasia, according to Jefferies. Shionogi has moved to acquire commercial-stage IntraBio for $2 billion upfront just weeks after the Texas-based biotech secured an FDA approval in a rare disease for which there are no other treatments. The centerpiece of the deal is IntraBio’s Aqneursa, which the FDA cleared in September 2024 for Niemann-Pick disease type C (NPC), an ultrarare, progressive genetic disorder that impacts lysosomal storage and can cause life-threatening complications. There are around 1,000 potential patients with NPC in the U.S., according to a Monday note from Jefferies. IntraBio’s net sales last year, all from Aqneursa in NPC, hit $68 million, according to a company release on Monday. Just a few weeks ago, IntraBio scored another rare disease approval for Aqneursa, expanding its use to patients with ataxia-telangiectasia (A-T), a prematurely fatal disease that involves the progressive degeneration of the cerebellum. The drug is the first and only treatment to receive approval for A-T worldwide. IntraBio continues to study Aqneursa in other rare diseases, including GM2 gangliosidoses, inherited cerebellar ataxias and CACNA1A-related disorders. The biotech, founded in 2015 , is also developing treatments for common disorders like migraine and dementias. Assuming that A-T has a similar patient population to NPC, “sales of several hundred million dollars should be achievable in the US alone,” Jefferies wrote, estimating $1 billion in global peak sales for the drug. Given this revenue potential, IntraBio’s $2 billion price tag “looks reasonable or slightly undervalued,” the analysts continued, while cautioning that efforts to bolster disease awareness to boost diagnoses “will incur costs.” For Shionogi, the IntraBio acquisition “offers readily achievable synergies and fits Shionogi’s growth strategy by strengthening its rare disease development capabilities,” Jefferies added. The Japanese drugmaker’s rare disease portfolio is currently anchored by the ALS drug Radicava. Shionogi acquired Radicava from Tanabe Pharma in December 2025 for $2.5 billion plus certain sales-based royalties. Beyond Radicava, Shionogi also touts rare disease pipeline programs in Fragile X syndrome and Jordan’s syndrome. Shionogi is actively seeking out M&A opportunities, chairperson and CEO Isao Teshirogi said in August. Aside from IntraBio and Tanabe, the Japanese pharma in May last year moved to absorb Japan Tobacco’s Torii Pharmaceutical for around $1 billion, gaining products such as the atopic dermatitis cream Vtama and the oral drug Miticure for dust mite allergies. Despite a slowdown in Q3 , 2026 is still shaping up to be a big year for M&A . Recent standout takeovers include Vertex Pharmaceuticals’ acquisition of Crinetics Pharmaceuticals—a deal originally billed to be worth $10 billion but which has been valued at $8.8 billion by S&P Capital IQ, after taking into account debt and other factors. Telix Pharmaceuticals also made waves last month after it revealed designs to absorb the radiopharma specialist ITM Isotope Technologies Munich for $1.65 billion upfront . Mergers & acquisitions M&A slows in Q3 but Vertex snags one of the year’s biggest in $10B Crinetics buy After a frantic second quarter, M&A steadied with just under $30 billion across 22 total transactions, according to a BioSpace analysis. October 1, 2026 · 1 min read Read more