Positive Topline Data Announced from Mirum’s LIVMARLI Phase 3 MARCH Study in Progressive Familial Intrahepatic Cholestasis (PFIC)

News | 10. 24. 2022

Mirum Pharmaceuticals

FOSTER CITY, CA--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced positive topline results from its pivotal Phase 3 MARCH study evaluating LIVMARLI® (maralixibat) oral solution in 93 patients with progressive familial intrahepatic cholestasis (PFIC) in a broad range of subtypes, age one to 17 years. The primary endpoint of improvement in pruritus severity in PFIC2 was statistically significant (p=0.0098).

“The advancement of new medications, particularly for rare diseases like PFIC, is incredibly important and provides hope to families who are in need of a treatment option that can effectively address itch, the most burdensome aspect of the disease”

The primary analysis was conducted in PFIC2 patients (n=31). The secondary analyses were evaluated in the All-PFIC cohort, which included PFIC2 as well as additional PFIC subtypes (n=64). The Full-Study population included All-PFIC as well as supplemental patients who had previously undergone surgery, had truncating mutations and other patients (n=93).