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Mirum Pharmaceuticals Initiates Rolling Submission of a New Drug Application for Maralixibat for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome and Launches Expanded Access Program
- Maralixibat would be the first treatment available for use in Alagille syndrome, if approved - Completion of the NDA submission expected in the first
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Maralixibat would be the first treatment available for use in Alagille syndrome, if approved\n- Completion of the NDA submission expected in the first quarter of 2021\n- Expanded Access Program for maralixibat now open\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 that it has submitted the first portion of its rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for maralixibat. Maralixibat is a novel, minimally absorbed, orally administered apical sodium dependent bile acid transporter (ASBT) inhibitor being evaluated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS). Mirum expects to complete the NDA submission in the first quarter of 2021.\n\n\nMirum also launched its Expanded Access Program (EAP) for maralixibat for the treatment of cholestatic pruritus in patients with ALGS one year of age and older. The EAP is open for registration in the United States and Canada. Through this program, physicians can request access to maralixibat for eligible patients who are not part of an ongoing clinical trial.\n\n\n“We are thrilled to initiate the rolling NDA submission for maralixibat, taking us one step closer to making this medicine widely available for patients with ALGS,” said Chris Peetz, president and chief executive officer at Mirum. “We believe the results of our clinical program demonstrate the potential of maralixibat to transform the treatment of this life-threatening disease. We are also pleased to launch our Expanded Access Program for patients with ALGS in the United States and Canada and are evaluating ways in which we can make maralixibat available for patients with ALGS in other countries. Additionally, we are planning to broaden access to maralixibat through our anticipated Marketing Authorization Application submission for patients with PFIC2 in Europe later this year.”\n\n\nAbout the NDA Submission\nMaralixibat was previously granted Rare Pediatric Disease Designation for ALGS and, as such, may qualify for receipt of a priority review voucher if the NDA is approved by the FDA. Maralixibat was also granted Breakthrough Therapy Designation for the treatment of pruritus...