武田薬品工業株式会社
Takeda today announced that it has submitted a new drug application to the Ministry of Health, Labour and Welfare（MHLW）for maralixibat chloride (development code: TAK-625, “maralixibat”), an ileal bile acid transporter (IBAT) inhibitor for the treatment of Alagille Syndrome (“ALGS”) and Progressive Familial Intrahepatic Cholestasis (“PFIC”).
The application is based on the results of Phase III clinical trials (TAK-625-3001 and TAK-625-3002) conducted in Japan for the treatment of ALGS and PFIC, as well as multiple clinical trials conducted overseas by Mirum.
In September 2021, Takeda entered into a licensing agreement with Mirum for the exclusive development and marketing rights of maralixibat in Japan.
Maralixibat is approved in September 2021 in the United States for treatment of cholestatic pruritus in patients with ALGS three months of age and older and has since been approved in several countries and regions around the world, including Europe. Maralixibat was approved in March 2024 in the United States for the treatment of cholestatic pruritus in patients with PFIC five years of age and older, and an application for the treatment of PFIC in patients three months and older in Europe has received positive opinion by Committee for Medicinal Products for Human Use (CHMP)1. The decision by the European Commission is expected by the third quarter of 2024.
Alagille syndrome (ALGS) is a rare genetic disorder characterized by a paucity of interlobular bile ducts, which eventually leads to biliary stasis and progressive liver dysfunction. The estimated incidence is 1 in 30,0002 people, and a national survey has put the figure at around 200-300 patients in Japan.3 ALGS can affect multiple organs, including the liver, heart, kidneys and central nervous system.4 Reports indicate that between 60% and 75% of ALGS patients need liver transplantation before reaching adulthood.5 Symptoms include jaundice, xanthomas and severe pruritus. The pruritus experienced by patients with ALGS is among the most severe in any chronic liver disease and is present in most affected children by the third year of life. 6
Progressive Familial Intrahepatic Cholestasis (PFIC) is a rare genetic disorder that impairs bile secretion from liver cells, causing an accumulation of bile in the liver cells and progressive liver disease. Symptoms typically appear in infancy and include severe pruritus, jaundice, impairment of growth and liver dysfunction. The disease is estimated to affect one in every 50,000 to 100,000 births in the United States and Europe. PFIC is designated as a chronic paediatric disease in Japan, but only about 2 to 8 new cases are registered annually with the “Medical Aid Program for Chronic Pediatric Diseases of Specified Categories”. PFIC has been reported to have multiple subtypes based on the gene responsible for the condition, and all subtypes share the common features of bile secretion impairment and progressive liver disease.
Maralixibat is an ileal bile acid transporter (IBAT) inhibitor suitable for oral administration and approved by the U.S. Food and Drug Administration (FDA) for the treatment of pruritus associated with biliary stasis in patients with Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). Moreover, it is the only IBAT inhibitor approved by the European Commission and Health Canada for the treatment of cholestatic pruritus in patients with ALGS. In addition, an application for approval of maralixibat in PFIC has received positive opinion by CHMP and favorable opinion by COMP recommending maintenance of Orphan Drug Designation. A decision by the European Commission is expected by the third quarter of 2024. Maralixibat has received Breakthrough Therapy designation for ALGS and PFIC2 (a subtype of PFIC) in the U.S. and Orphan Drug designation for ALGS and PFIC in the U.S. and Europe. In Japan, the Ministry of Health, Labour and Welfare granted Orphan Drug designation for the expected indications of ALGS and PFIC on December 16, 2022.
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CHENODAL® (chenodiol) tablets.
LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (three months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients five years of age and older.
To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X (Twitter).
Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare disease, plasma-derived therapies, neuroscience, three oncology and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit www.takeda.com.
We believe that Gastroenterology (GI) and liver diseases are not just life-disrupting conditions, but diseases that can impact a patient’s quality of life. Beyond a fundamental need for effective treatment options, we understand that improving patients’ lives also depends on their needs being recognized. With over 35 years of experience in gastroenterology, Takeda has made significant strides in addressing GI patient needs with treatments for inflammatory bowel disease (IBD), acid-related diseases, short bowel syndrome (SBS), and motility disorders. We are making significant strides toward closing the gap on new areas of unmet needs for patients who have celiac disease, eosinophilic esophagitis, alpha-1 antitrypsin-associated liver disease and Crohn’s disease, among others. Together with researchers, patient groups and more, we are working to advance scientific research and clinical medicine in GI.
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