Mirum Pharmaceuticals Announces Health Canada Authorization of LIVMARLI® for the Treatment of Cholestatic Pruritus in Patients with Progressive Familial Intrahepatic Cholestasis (PFIC)
Mirum Pharmaceuticals, Inc. a leading rare disease company, today announced that Health Canada has authorized LIVMARLI® (maralixibat) oral solution and tablet formulation for the treatment of cholestatic pruritus in patients aged 12 months or older with progressive familial intrahepatic cholestasis (PFIC). PFIC is a rare, inherited pediatric liver disease caused by impaired bile flow, leading to the accumulation of bile acids in the liver and bloodstream. Cholestasis in PFIC is associated with severe pruritus that can significantly impede quality of life for patients and caregivers. LIVMARLI is available in Canada for the treatment of PFIC as a 19 mg/mL oral solution and as a tablet formulation for patients weighing 22 kg or more who can swallow tablets.
“Health Canada’s authorization of LIVMARLI for the treatment of cholestatic pruritus in patients with PFIC aged 12 months and older has the potential to be transformational for patients living with the severe burden of symptoms associated with this disease,” said Jamie Twiselton, General Manager, Mirum Pharmaceuticals Canada. “The authorization of both the 19 mg/mL oral solution and the tablet formulation of LIVMARLI offers meaningful flexibility and convenience to support treatment as patients with PFIC age.
Health Canada’s authorization of LIVMARLI for the treatment of cholestatic pruritus in PFIC is supported by data from the Phase 3 MARCH study, the largest randomized trial conducted in PFIC, which enrolled 93 patients, including patients from Canada. The MARCH study included patients across a range of genetic PFIC types, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 and unidentified mutational status. In the MARCH study, patients treated with LIVMARLI demonstrated a highly statistically significant (p<0.0001) reduction in pruritus severity versus placebo.
“PFIC is a complex condition to treat, and pruritus is often among the most burdensome symptoms for children and families,” said Susan M. Gilmour, MD, MSc, FRCPC, Professor Pediatric Gastroenterology/Nutrition, Department of Pediatrics, Faculty of Medicine and Dentistry, University of Alberta, Stollery Children’s Hospital. “An authorized option in Canada that addresses cholestatic pruritus and is indicated across multiple PFIC types, while offering flexibility in formulation, represents an important advancement for clinical care.
Patients and families affected by PFIC know how all-consuming cholestatic pruritus can be,” said Emily Ventura, Co-Founder and Executive Director of the PFIC Network. “For many patients, the itch is so severe it impacts sleep, nutrition and child development. Health Canada’s authorization of LIVMARLI offers hope for meaningful relief from pruritus, and the availability of both liquid and tablet formulations offers treatment flexibility for patients.”
LIVMARLI® is also authorized in Canada for the treatment of cholestatic pruritus in patients 12 months of age or older with Alagille syndrome (ALGS).
About PFIC
Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease, typically leading to liver failure. In people with PFIC, liver cells are less able to secrete bile. The resulting buildup of bile causes liver disease in affected individuals. Signs and symptoms of PFIC typically begin in infancy. Patients experience severe itching, jaundice, failure to grow at the expected rate (failure to thrive) and an increasing inability of the liver to function (liver failure). The disease is estimated to affect one in every 50,000 to 100,000 births in Canada. Many types of PFIC have been genetically identified, all of which are similarly characterized by impaired bile flow and progressive liver disease.
About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets
LIVMARLI® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. LIVMARLI is authorized in Canada for the treatment of cholestatic pruritus in patients aged 12 months and older with ALGS.
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