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Mirum Pharmaceuticals Announces Completion of Rolling NDA Submission for Maralixibat in Alagille Syndrome
- Maralixibat U.S. launch expected in second half of 2021, if approved FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM)
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Maralixibat U.S. launch expected in second half of 2021, if approved\n\n FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced it has completed submission of its rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older. ALGS is a rare liver disease for which there are currently no approved therapies. The company is planning for the U.S. launch of maralixibat in the second half of 2021, should the medication be approved by the FDA for this indication.\n\nThe NDA submission included the results of the ICONIC study and additional supportive studies totaling 86 patients with ALGS with up to six-year follow-up. The clinical safety data submitted for maralixibat includes over 1,600 individuals treated.\n\n“Patients, families and physicians have an urgent need for effective medications to address the severe and life-altering symptoms associated with ALGS,” said Chris Peetz, president and chief executive officer at Mirum. “This submission brings us one step closer to a potential approval of maralixibat and providing a much-needed treatment option for the patients and their families suffering with ALGS.”\n\nUntil maralixibat is approved by the FDA for prescription, maralixibat is available to eligible patients with ALGS through Mirum’s Expanded Access Program (EAP). The EAP is available in the United States, Canada, Australia and in regions throughout Europe. 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 Clinicaltrials.gov. Physicians in the United States and Canada who would like to request access for their patients can email [email protected]. Physicians in Europe and Australia can contact [email protected] with a reference line of “Mirum ALGS EAP request”.\n\nMaralixibat was granted Rare Pediatric Disease Designation for ALGS and, as such, may qualify for receipt of a priority review voucher, if 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...