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－Ministry of Health, Labour and Welfare grants Orphan Drug Designation to Maralixibat for Alagille syndrome and progressive familial intrahepatic cholestasis.
OSAKA, Japan, December 19, 2022 – Takeda (TSE:4502/NYSE:TAK) announced that it has received Orphan Drug Designation from the Ministery of Health, Labour and Welfare for Maralixibat chloride (TAK-625) for the expected indications of Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).
ALGS is a very rare, progressive, and severe disease. Symptoms include stunted growth and severe pruritus. PFIC is a very rare, progressive, and serious disease caused by bile stasis in hepatocytes due to various genetic abnormalities. Both ALGS and PFIC are designated as specific pediatric chronic diseases and designated as intractable diseases. Maralixibat is currently in phase 3 clinical trials in Japan for the treatment of ALGS and PFIC.
Maralixibat is being developed by Mirum Pharmaceuticals, Inc. Mirum and Takeda entered into an exclusive licensing agreement for the development and commercialization of maralixibat in Japan, in September 2021.
Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. Maralixibat inhibits the ileal bile acid transporter (IBAT), resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated effects.
Drugs are given orphan drug designation by the Ministry of Health, Labour and Welfare upon consultation with the Pharmaceutical Affairs and Food Sanitation Council in accordance with Article 77-2, Paragraph 1 of the Pharmaceuticals and Medical Devices Act if they meet all of the following criteria.
Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetics and Hematology, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people’s lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions. For more information, visit https://www.takeda.com.
The drug information contained herein is intended to disclose corporate information. Nothing contained in this document should be considered a solicitation, promotion, or indication for any prescription drug, including those currently under development.