The European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion of maralixibat oral solution, Livmarli®, to treat cholestatic pruritus in patients with Alagille syndrome (ALGS) aged 2 months and older, according to a news release from Mirum Pharmaceuticals, Inc.
The CHMP positive opinion is the scientific recommendation to the European Commission for grant marketing authorization in Europe. Maralixibat oral solution is currently approved in the United States for cholestatic pruritus in patients with ALGS aged 1 year and older.
The maralixibat oral solution produced by the company has received praise from CHMP regarding its use in treating patients with ALGS who experience cholestatic pruritus. This is highly significant, given that CHMP can make recommendations to the European Commission to allow marketing authorization in Europe. If approved, this drug will be the first and only approved treatment in Europe for the effects of ALGS.
“We are excited about the potential approval and availability of Livmarli for patients in Europe,” said Professor Emmanuel Jacquemin, MD, PhD, head of pediatric hepatology and liver transplantation unit, Bicêtre Hospital, AP-HP, Paris-Saclay University, Le Kremlin-Bicêtre, France.
Read more about ALGS etiology
In addition, the Israeli Ministry of Health approved this drug to treat patients aged 1 year and older with ALGS suffering from cholestatic pruritus. This means that the drug is ready for global expansion outside the United States.
Maralixibat oral solution is currently being tested in late-stage clinical trials to treat other rare cholestatic liver diseases such as biliary atresia and progressive familial intrahepatic cholestasis. It has received orphan designation for the former two diseases, as well as for ALGS.
ALGS is a rare genetic disorder in which bile duct abnormalities often progresses to liver failure. Bile duct structural abnormalities cause the retention of bile acids, leading to cholestasis and liver inflammation. Debilitating pruritus is a sign that the disease has advanced to a stage in which liver transplantation may be needed.
Mirium Pharmaceuticals announces positive CHMP opinion for LIVMARLI® (maralixibat) oral solution for cholestatic pruritus in patients with Alagille syndrome two months of age and older. News release. Mirium Pharmaceuticals, Inc.; October 14, 2022.