Japan sets eyes on rare pediatric liver diseases

US-based Mirum Pharmaceuticals, Inc. and Japanese firm Takeda Pharmaceutical have entered into an exclusive licensing agreement for the development and commercialization of maralixibat in Japan for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA). Maralixibat, an investigational, orally administered medication, is being evaluated globally in ALGS, PFIC, and BA.

Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. 

Under the terms of the agreement, Takeda will be responsible for regulatory approval and commercialization of maralixibat in Japan. Takeda will also be responsible for development, including conducting clinical studies in cholestatic indications.

“There is a significant unmet medical need for a treatment to help patients with cholestatic diseases such as ALGS and PFIC in Japan and developing novel treatment for those patients suffering from rare liver diseases is a top priority for Takeda’s global R&D strategy,” said Dr. Naoyoshi Hirota, general manager of Takeda development center Japan.