FOSTER CITY, Calif.– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced two global advancements for LIVMARLI® (maralixibat) oral solution, an oral minimally absorbed ileal bile acid transporter (IBAT) inhibitor currently approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.
The European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion of LIVMARLI for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) two months of age and older. The CHMP positive opinion is the scientific recommendation to the European Commission for grant marketing authorization in Europe. LIVMARLI was previously approved by the U.S. Food and Drug Administration in 2021 as the first and only treatment for cholestatic pruritus in patients with ALGS one year of age and older.
Mirum also announced that its partner, Neopharm, received commercial and marketing authorization by the Israeli Ministry of Health, Israel’s regulatory agency responsible for the approval of new medicines, granting approval of LIVMARLI for the treatment of cholestatic pruritus in patients with ALGS one year of age and older.
“LIVMARLI is the first and only medicine to receive CHMP positive opinion to address cholestatic pruritus in patients with Alagille syndrome and we are hopeful that this medication provides a meaningful treatment option for people suffering from this devastating disease,” said Chris Peetz, president and chief executive officer at Mirum. “Our experience with LIVMARLI in the U.S. has shown the impact it can have on the lives of patients and their families living with ALGS. We are eagerly preparing for the launch of LIVMARLI in Europe and are excited about the opportunity to further expand LIVMARLI’s availability globally, which is off to a great start with the recent approval in Israel.”
ALGS is a rare genetic disease caused by abnormalities in bile ducts that can lead to progressive liver disease. Malformed or reduced bile ducts cause retention of bile acids in the liver leading to cholestasis and ultimately inflammation and liver injury which prevents the liver from working properly. Cholestasis in ALGS leads to a debilitating pruritus impacting the quality of life so significantly that pruritus is a leading indication for liver transplantation.
The positive CHMP 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. Data from ICONIC demonstrated statistically significant and clinically meaningful reductions in pruritus compared to placebo with a mean difference of -1.4 points between groups, as well as significant reductions in serum bile acids, both of which were durably maintained over several years of treatment. The proposed label also includes reductions in xanthoma severity.
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 Alagille syndrome, a rare and devastating liver disease impacting 1 out of 30,000 people globally.
“The positive opinion adopted by the CHMP is an important step forward in the treatment of cholestatic pruritus for people living with Alagille syndrome, given the lack of treatments to effectively address this terrible disease burden which can ultimately lead to a decision for liver transplantation,” 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. “We are excited about the potential approval and availability of LIVMARLI for patients in Europe.”
“Alagille syndrome is seen in patients globally. Outside the U.S. only limited treatment options have been available until now to effectively help people deal with the debilitating effects of the disease, especially pruritus. Pruritus causes significant disruptions to the lives of both patients and their family members,” said Roberta Smith, president of the Alagille Syndrome Alliance. “We are thrilled that there is hope on the near horizon for people with Alagille syndrome in Europe and are optimistic that LIVMARLI may provide relief to those suffering from cholestatic pruritus in ALGS if approved, much like we’ve heard from our U.S. families following LIVMARLI’s launch in the United States.”
