LIVMARLI Approved in Japan for ALGS and PFIC

Mirum Pharmaceuticals, Inc. has announced that Takeda Pharmaceutical Company Limited, its partner, has secured approval from Japan's Ministry of Health, Labour, and Welfare for the medication LIVMARLI® (maralixibat) oral solution. This approval is for the treatment of cholestatic pruritus in patients affected by Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) in Japan. LIVMARLI is now recognized as the sole treatment option available in Japan for individuals suffering from pruritus associated with these rare liver conditions.

Chris Peetz, the CEO of Mirum, expressed his enthusiasm about this development, noting Takeda's reputation as a leading global pharmaceutical entity excelling in the development and marketing of medications for rare diseases. He conveyed confidence that under Takeda’s management, LIVMARLI could significantly improve the lives of ALGS and PFIC patients in Japan.

The partnership between Mirum and Takeda was established in September 2021 through an exclusive license agreement. This agreement aimed at the development and commercialization of LIVMARLI for rare cholestatic pediatric liver diseases within Japan. As per the agreement's terms, Mirum stands to receive a share of Takeda’s annual net sales derived from LIVMARLI.

LIVMARLI has achieved approval for the treatment of pruritus linked to ALGS in over 40 countries and for PFIC in more than 30 nations globally. The medication is an orally administered ileal bile acid transporter (IBAT) inhibitor sanctioned by the U.S. FDA and the European Medicines Agency for two pediatric cholestatic liver diseases. In the U.S., it is approved for treating cholestatic pruritus in patients with ALGS from three months of age and PFIC from twelve months of age. Similarly, in Europe, it is approved for ALGS patients from two months of age and for PFIC from three months of age.

LIVMARLI is also being examined in additional settings for cholestatic pruritus through the Phase 3 EXPAND study. This study aims to explore further applications and benefits of the medication in managing cholestatic pruritus.

Mirum Pharmaceuticals is a biopharmaceutical firm committed to revolutionizing the treatment of rare diseases that affect both children and adults. The company’s portfolio includes three approved medications: LIVMARLI, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for treating two rare liver diseases affecting both children and adults and is currently available in multiple countries, including the U.S. and Europe, for specific age groups.

Besides LIVMARLI, Mirum is progressing with its late-stage pipeline, which features two investigational treatments for rare diseases. One such treatment, Volixibat, is under evaluation in two potentially registrational studies: the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and the Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has received Breakthrough Therapy Designation for treating cholestatic pruritus in patients with primary biliary cholangitis. Furthermore, Mirum is preparing for a Phase 2 study of MRM-3379, a PDE4D inhibitor for Fragile X syndrome, a rare genetic neurocognitive disorder.

Through these efforts, Mirum Pharmaceuticals is advancing its mission to deliver innovative solutions for rare diseases, improving the quality of life for affected individuals worldwide.