Shionogi Expands Rare Disease Presence with Acquisition of ALS Therapy in $2.5B Deal

Rare Daily Staff

Shionogi said it would acquire for $2.5 billion a newly established company that Tanabe Pharma will create to hold the rights to Radicava ORS, a U.S. Food and Drug Administration-approved therapy for the rare neurodegenerative disease amyotrophic lateral sclerosis, or ALS.​

As part of the transaction, Shionogi will own the new business, which will become a wholly owned subsidiary of Shionogi. In addition to Radicava ORS, it will include IV Radicava. Shionogi may also pay a royalty on future sales, subject to certain conditions.​

The acquisition is driven by Shionogi’s strategic push into rare diseases, which it views as high social impact, quality-of-life disorders. The company has made these conditions a priority area for research and development. As part of those efforts, Shionogi is establishing a presence in rare disease through existing research programs in Fragile X syndrome, Jordan’s syndrome, and Pompe disease, in addition to potential early phase rare disease assets that recently became part of Shionogi’s pipeline. The Radicava acquisition creates the commercial infrastructure to accelerate delivery of new rare disease medicines once regulatory approvals are obtained.​

ALS is characterized by the selective degeneration and loss of motor neurons, leading to muscle weakness and respiratory impairment. After onset, muscle atrophy gradually progresses, and ultimately respiratory muscle paralysis severely impairs life expectancy. Multiple factors, including oxidative stress and glutamate-induced excitotoxicity, are involved in its pathophysiology, and treatment options to slow disease progression remain extremely limited.​

Radicava (edaravone) was discovered and developed for ALS by Mitsubishi Tanabe Pharma, which began researching ALS in 2001 through an iterative clinical platform over a 13-year period. In 2015, edaravone was approved as Radicut for the treatment of ALS in Japan and South Korea, and it subsequently received marketing authorizations in Canada, Switzerland, China, Indonesia, Thailand, Malaysia, Australia, Brazil, and the United States. Marketing authorization for Radicava oral suspension was granted in Canada and Switzerland, and Radicut oral suspension 2.1 percent was granted regulatory approval in Japan.​

“This planned acquisition will solidify our strategic focus in rare disease and immediately add capabilities to ensure long-term success in this important category,” said Nathan McCutcheon, president and CEO of Shionogi Inc. “Going forward, this infrastructure will support future launches in rare disease, including Shionogi’s development programs in Fragile X syndrome, Jordan’s syndrome, and Pompe disease.”