Maralixibat (TAK-625) for Rare Cholestatic Liver Diseases Designated as an Orphan Drug

－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.

About Maralixibat

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.

About Orphan Drugs

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.

• There must be fewer than 50,000 patients in Japan for whom the drug is intended. However, if the drug is for a designated intractable disease, the number of patients is instead limited to the number specified in Article 5, Paragraph 1 of the Intractable Diseases Act (approximately one-thousandth of the population).
• In addition to being indicated for serious illnesses, there should be no appropriate alternative drug or treatment, or the product should have a particularly high medical value, such as being expected to have a significantly higher level of efficacy or safety than existing drugs.
• There should be a theoretical rationale for the use of the product for the target disease, and the development plan should be appropriate.

About Takeda Pharmaceutical Company Limited

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.

Disclaimer

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.

Media Contacts:
Tatsuhiro Kanoo
[email protected]
＋81 3-3278-3634

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