News

Mirum Pharmaceuticals Announces Positive Phase 3 RESTORE Study Results Evaluating Chenodal in Patients with Cerebrotendinous Xanthomatosis

Oct 02, 2023 – Primary endpoint met (p<0.0001); 20-fold difference in urine bile alcohols – First and only prospective, controlled clinical trial in CTX – Plasma cholestanol endpoint met (p=0.0083), the driver of poor outcomes in CTX patients – Data to be submitted for presentation at future medical congress – Mirum plans to file an NDA with the U.S. FDA…

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Mirum Pharmaceuticals Announces New PDUFA Date for LIVMARLI for the Treatment of Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis

Oct 17, 2023 FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has extended the review of the Supplemental New Drug Application (sNDA) for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC). The new Prescription Drug…

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Mirum Pharmaceuticals Announces Enrollment Completion in Phase 2b EMBARK Biliary Atresia Study

May 11, 2023 FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced it has completed enrollment of its Phase 2b EMBARK study for pediatric patients with biliary atresia, the most common indication for pediatric liver transplant. The EMBARK study is a global, double-blind, randomized, placebo-controlled, 26-week trial evaluating the efficacy and safety of LIVMARLI®…

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Mirum Announces Label Expansion for LIVMARLI in the United States to Include Infants Three Months of Age and Older

– LIVMARLI immediately available for prescribing for patients three months of age and older FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved a reduction in age from one year to three months for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in…

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Minoryx presents positive interim results from NEXUS registration study for leriglitazone targeting pediatric ALD patients with cerebral adrenoleukodystrophy (cALD)

2023-04-25 Initial evidence of disease stabilization was demonstrated for all patients using radiological, clinical and plasma biomarker assessments at 6 months (24 weeks) of treatment Mataró, Barcelona, Spain, April 25, 2023 – Minoryx Therapeutics, a registration stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces 24-week interim results…

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Minoryx gains FDA approval to initiate a Phase 3 clinical trial in patients with cerebral Adrenoleukodystrophy

2023-05-31 US trial with leriglitazone to commence in mid-2023 providing an FDA-approved route to US market whilst EMA evaluates European Marketing Authorization Application (MAA) Mataró, Barcelona, Spain, May 31, 2023 – Minoryx Therapeutics, a registration stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces the US Food and Drug Administration (FDA) approval…

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