Mirum Pharma: LIVMARLI Gets Positive CHMP Opinion For Use In Two Months Of Age And Older

(RTTNews) - Mirum Pharmaceuticals, Inc. (MIRM) announced Friday that the European Committee for Medicinal Products for Human Use or CHMP has adopted a positive opinion of LIVMARLI (maralixibat) oral solution to treat cholestatic pruritus in patients with Alagille syndrome or ALGS two months of age and older.

The company also announced that its partner, Neopharm, received commercial and marketing authorization by the Israeli Ministry of Health, granting approval of LIVMARLI to treat cholestatic pruritus in patients with ALGS one year of age and older.

ALGS is a rare genetic disease caused by abnormalities in bile ducts that can lead to progressive liver disease. The company noted that Cholestasis in ALGS leads to a debilitating pruritus impacting the quality of life so significantly that pruritus is a leading indication for liver transplantation.

LIVMARLI is an oral minimally absorbed ileal bile acid transporter or IBAT inhibitor currently approved in the U.S. for the treatment of cholestatic pruritus in patients with ALGS one year of age and older.

The company noted that the CHMP positive opinion is the scientific recommendation to the European Commission for granting marketing authorization in Europe. The European Commission's endorsement of the CHMP positive opinion is expected in the fourth quarter of 2022.

If approved in Europe, LIVMARLI would be the first and only approved treatment to address the effects of ALDS.

The positive opinion is based on data from the pivotal ICONIC study including five years of data across the LIVMARLI clinical program resulting in a robust body of evidence in patients with ALGS. The trial data showed statistically significant and clinically meaningful reductions in pruritus compared to placebo.

Mirum noted that it is currently evaluating LIVMARLI in late-stage clinical studies in other rare cholestatic liver diseases including progressive familial intrahepatic cholestasis or PFIC and biliary atresia. The drug has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia.

For More Such Health News, visit rttnews.com