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…
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…
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®…
– 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…
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…
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…
