News

2025-07-17 Barcelona, Spain, 17 July 2025 – Minoryx Therapeutics, a late-stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders today announces that it has secured a EUR 26.9m non-dilutive grant for the development of its LERI4CNS project, which will validate leriglitazone for treating potentially lethal rare diseases affecting the…

– 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…

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®…

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…

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…

Apr 12, 2023 FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced estimated revenue, including net product sales, and cash balance for the first quarter 2023. The company’s preliminary unaudited financial results for the first quarter of 2023 include: Additionally, the company updated its guidance for the VISTAS Phase 2b clinical trial evaluating volixibat…