RARE Daily

Mirum and Takeda Partner to Develop and Commercialize Rare Pediatric Liver Disease Drug in Japan

September 21, 2021

Mirum Pharmaceuticals and Takeda Pharmaceutical entered into an exclusive licensing agreement for the development and commercialization of maralixibat in Japan as a treatment for rare cholestatic pediatric liver diseases.

Photo: Chris Peetz, president and chief executive officer of Mirum

“Takeda is a leading global biopharmaceutical company with extensive experience in development and commercialization of novel therapies to treat rare diseases as well as gastroenterology and hepatology, making them an ideal partner as we look to accelerate the delivery of maralixibat to children living with rare liver diseases in Japan,” said Chris Peetz, president and chief executive officer of Mirum. “As we approach potential commercialization in the United States and complete the recent filing for Alagille syndrome in Europe, our goal is to partner with top companies outside of North America and Europe to ensure global reach for patients with these terrible 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 Naoyoshi Hirota, general manager of Takeda development center Japan. “This agreement reinforces Takeda’s commitment to developing highly differentiated medicines to improve the health and quality of life of patients.”

Maralixibat is an experimental, orally administered medication that is being evaluated in the rare liver diseases Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA). It targets the apical sodium dependent bile acid transporter (ASBT), ultimately resulting in lower levels of bile acid systemically, which could mediate liver damage. If left untreated, these conditions can lead to liver failure and inflict life-altering symptoms including stunted growth and severe, non-stop itching. Under current standard of care, patients often require a liver transplant and are at higher risk of many serious health issues.

Mirum’s new drug application to the U.S. Food and Drug Administration for maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome is currently under priority review with an FDA decision date of September 29, 2021. Mirum also recently submitted a Marketing Authorization Application to the European Medicines Agency for maralixibat for the treatment of cholestatic liver disease in patients with ALGS.

Author: Rare Daily Staff

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