Mirum Pharmaceuticals’ LIVMARLI (maralixibat oral solution) Receives Positive Reimbursement Recommendation by Canada’s CADTH for Patients with Cholestatic Pruritus in Alagille Syndrome

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) has recommended public reimbursement for LIVMARLI (maralixibat oral solution) for the treatment of cholestatic pruritus in patients with Alagille Syndrome (ALGS), with certain conditions. This announcement follows Health Canada’s authorization of LIVMARLI in this indication in 2023.

“The positive reimbursement recommendation for LIVMARLI by CADTH is an important step forward for patients suffering from the debilitating effects of cholestatic pruritus related to Alagille syndrome,” said Chris Peetz, chief executive officer at Mirum. “Accelerating access in all provinces to this meaningful treatment is critical as it is the first and only approved therapy available in this setting in Canada. These patients suffer greatly from the debilitating and disruptive effects of Alagille syndrome, which often starts in early childhood.”

“CADTH’s recommendation of reimbursement for LIVMARLI provides a meaningful advancement in the treatment of cholestatic pruritus for patients with Alagille syndrome. I am pleased that LIVMARLI will now become more accessible in Canada, where I have seen the critical need for a treatment to address the itch caused by cholestasis,” said Dr. Binita Kamath, Director, Hepatology Program and Senior Associate Scientist at The Hospital for Sick Children (SickKids), Toronto, Canada.

“This recommendation is incredibly meaningful as it signifies hope for patients and their families who have suffered from the disruptive and painful effects of pruritus for far too long,” said Roberta Smith, president, Alagille Syndrome Alliance and mother of a daughter with ALGS. “We are grateful to CADTH for ensuring support of this new and important medication.”

The CADTH recommendation is based on data from the pivotal ICONIC study including six years of data across the LIVMARLI clinical program resulting in a robust body of evidence in patients with cholestatic pruritus in ALGS. Data from ICONIC demonstrated statistically significant and clinically meaningful reductions in pruritus compared to placebo, as well as significant reductions in serum bile acids, both of which were durably maintained over several years of treatment.