RARE Daily

Canada Approves Mirum’s Livmarli for Cholestatic Pruritus in Patients with Alagille Syndrome

July 25, 2023

Rare Daily Staff

Health Canada has authorized the use of Mirum Pharmaceuticals’ Livmarli for the treatment of cholestatic pruritus in patients with Alagille syndrome.

Alagille syndrome (ALGS) is a rare genetic disorder caused by abnormalities in bile ducts that can lead to progressive liver disease. Malformed or reduced bile ducts cause cholestasis, the accumulation of bile acids in the liver, which leads to inflammation and liver injury, and prevents the liver from working properly. Cholestasis in ALGS is associated with pruritus which is among the most common indications for liver transplant in ALGS.

“Today’s approval in Canada furthers our commitment to ensuring patients globally have a meaningful and effective treatment option for the unrelenting burden of cholestatic pruritus impacting patients and their families living with ALGS,” said Chris Peetz, president and CEO at Mirum.

“The years of data collected have shown that Livmarli can provide tremendous benefit, potentially giving many patients the opportunity to live transplant-free,” said Binita Kamath, Hospital for Sick Children, Toronto, Canada. “The statistical robustness of results seen in the Livmarli studies give confidence that this treatment has the potential to have meaningful outcomes for patients with debilitating pruritus.”

The Health Canada authorization 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.

Results from these studies showing robust and meaningful improvements for patients with cholestatic pruritus associated with ALGS were featured in several publications, including The Lancet and Hepatology.

Livmarli oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor and the only approved medication by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome three months of age and older.

In addition to today’s authorization, Livmarli is approved in the United States for the treatment of cholestatic pruritus in patients with ALGS three months and older and in Europe in the same indication, two months of age and older. Mirum has submitted a supplemental new drug application to the FDA for approval of Livmarli in cholestatic pruritus associated with progressive familial intrahepatic cholestasis (PFIC) in patients three months and older, and to the EMA for the treatment of PFIC in patients two months of age and older. Both submissions are currently under regulatory review. Regulatory submissions outside of the United States, Canada, and Europe are being pursued by partners and distributors in key markets globally.

Livmarli is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including biliary atresia. Livmarli has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia.

Photo: Chris Peetz, president and CEO at Mirum

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