Livmarli approved in the European Union for patients with progressive familial intrahepatic cholestasis

The approval follows a positive opinion by the CHMP which concluded that Livmarli in PFIC brings significant clinical benefit based on improved efficacy and a major contribution to patient care compared to the existing approved treatment for PFIC. Further, evaluation by COMP recommended maintenance of Orphan Drug Designation for LIVMARLI in PFIC.

The approval is based on data from the Phase III MARCH study, the largest randomized trial conducted in PFIC, with 93 patients across a range of genetic PFIC types, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6, and unidentified mutational status. Data from MARCH showed statistically significant reduction in pruritus (p<0.0001) and serum bile acids (p><0.0001) between livmarli versus placebo in the all-pfic cohort (n="64)." significant improvements were also observed in total bilirubin and growth versus placebo. the most common treatment emergent adverse event was diarrhea, which was predominantly mild, with no severe cases, and transient.

"Livmarli's approval provides a treatment backed by years of clinical research and meaningful data that demonstrate a reduction in cholestatic itch and prognostic markers of improved liver health, including a reduction of serum bile acids, all important signals for positive long-term outcomes,” said Professor Richard Thompson, King’s College London. “It is encouraging to know that physicians in Europe will have a new option that has the potential to improve the liver health and quality of life for patients and their families.”

Livmarli is also approved by the FDA for the treatment of cholestatic pruritus in patients with PFIC five years of age and older. Mirum has submitted a supplemental new drug application to introduce a higher concentration formulation of Livmarli, used during the MARCH study, to enable label expansion for younger patients with PFIC, and expects to receive FDA feedback this year.