Zatolmilast

Japanese drugmaker Shionogi announced Monday that it will be taking ownership of one of the only approved ALS drugs in a multibillion-dollar deal, with implications for its broader ambitions in rare disease. The company will pay $2.5 billion to acquire both the oral and IV formulations of Radicava, also known as edaravone, from Bain Capital-owned Tanabe Pharma. As part of the deal, Tanabe will spin out a newco for all Radicava operations, which will operate as a subsidiary of Shionogi. For Shionogi, the deal represents a significant investment in the rare disease space. The company is researching drugs for rare genetic disorders, most of which are in early- or mid-stage testing. But it also has zatolmilast, a PDE4D inhibitor that is in Phase 3 for the inherited developmental disorder known as fragile X syndrome. The acquisition of Radicava suggests Shionogi may want to build out its commercial infrastructure in rare disease ahead of zatolmilast’s first pivotal readouts. A Shionogi spokesperson declined to comment directly on its future plans, but said in an emailed statement that the deal “will solidify our strategic focus in rare disease and immediately add capabilities to ensure long term success.” As part of the transaction, Tanabe may receive royalties on future sales, the companies said in the announcement . Intravenous Radicava costs about $160,000 per year, and the oral formulation costs about $175,000. ALS is universally fatal. For years, it has been one of drug development’s most difficult fields, with most efforts failing. Radicava is one of the only exceptions, reaching the market in Japan in 2015 before an FDA approval in 2017. It is one of two FDA-approved drugs for all forms of ALS, along with riluzole, an oral drug from the French drugmaker Rhône-Poulenc (later acquired by Sanofi) that was approved in the 1990s. But both treatments provide only modest benefits, typically slowing ALS progression by a few months. Biogen and Ionis also got their drug Qalsody approved in 2023, but only for a genetic form of ALS making up about 2% of all diagnoses. There remains a significant demand for new ALS treatments, and the patient community is highly organized. A first-of-its-kind basket study for experimental ALS drugs is ongoing at Massachusetts General Hospital, but it’s seen limited success . Additionally, in 2022, multiple advocacy groups successfully put pressure on the FDA to be more flexible for ALS treatments, as the agency granted a full approval to Amylyx Pharmaceuticals’ Relyvrio despite an ongoing Phase 3 study. However, that trial ultimately failed , and Relyvrio was withdrawn.