Business
Mirum Pharmaceuticals Broadens Expanded Access Program for Maralixibat in Alagille Syndrome to Europe and Australia
Maralixibat Expanded Access Program now available for patients with pruritus associated with Alagille syndrome in Australia and 10 countries in Europe.
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n\nMaralixibat Expanded Access Program now available for patients with pruritus associated with Alagille syndrome in Australia and 10 countries in Europe.\n\n\n FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced it has broadened its Expanded Access Program (EAP) for maralixibat for the treatment of patients with cholestatic pruritus associated with Alagille syndrome (ALGS) to Australia and countries in Europe. The announcement follows the Company’s recent initiation of an EAP in the United States and Canada for maralixibat in the same indication.\n\n“Maralixibat has the potential to make a meaningful difference in the treatment of Alagille syndrome and it is our goal to help ensure that eligible patients have access to maralixibat until it is available for prescribing in Europe and Australia,” said Chris Peetz, president and chief executive officer at Mirum. “Continuity of treatment is critically important for children with rare diseases, like Alagille syndrome, and we hope that early access to investigational treatments such as maralixibat will help to reduce many of the debilitating symptoms associated with the disease as well as the need for liver transplantation.”\n\nMaralixibat is a novel, minimally absorbed, orally administered apical sodium dependent bile acid transporter inhibitor being evaluated for the treatment of cholestatic pruritus in patients with ALGS. Mirum initiated its rolling New Drug Application (NDA) for the treatment of cholestatic pruritus in patients with ALGS to the U.S. Food and Drug Administration (FDA) in September 2020, expects to complete the submission in the first quarter of 2021, and is preparing for a U.S. commercial launch in the second half of 2021.\n\nAbout the Maralixibat Expanded Access Program \n\nThe EAP, sometimes referred to as “compassionate use,” provides a potential pathway for a patient with an immediately life-threatening condition or serious disease to gain access to an investigational medicine for the treatment of that disease outside of a clinical trial when no comparable or satisfactory alternative therapy options are available.\n\nThe goal of Mirum’s EAP is to provide access to maralixibat for the tr...