Mirum 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.
Mirum 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.
“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.”
About the NDA Submission
Maralixibat 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 associated with ALGS in patients one year of age and older. Maralixibat was granted Orphan Drug Designation by the FDA for the treatment of patients with PFIC and ALGS in the United States.
Data from the maralixibat Phase 2 ICONIC study evaluating patients with ALGS serves as the basis of efficacy for the submission. Previously presented data from this study in November 2019 are available within the Publications and Presentations section on Mirum’s website.
Mirum expects to complete the rolling NDA submission in the first quarter of 2021 and is planning for a potential launch of maralixibat in ALGS in the second half of 2021.
About the Expanded Access Program
The 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.
The goal of Mirum’s EAP is to provide access to maralixibat for the treatment of cholestatic pruritus in eligible patients with ALGS prior to FDA approval of the medication and until maralixibat is available by prescription.
Requests for expanded access to maralixibat must be made by a licensed physician. Physicians and patients can learn more about the maralixibat EAP by visiting the program website at www.ALGSEAP.com or via https://clinicaltrials.gov/ct2/show/NCT04530994. Physicians who would like to request access for their patient can contact MirumALGS@clinigengroup.com.
For patients with PFIC, access to maralixibat is possible through our MARCH Phase 3 study, which is currently open to enrollment. More information can be found at: https://pfictrial.com. Mirum plans to continually evaluate the need for expanded access to maralixibat as studies reach enrollment milestones.
About Maralixibat
Maralixibat is a novel, minimally absorbed, orally administered investigatio...
(All Rights Reserved)