Mirum Pharmaceuticals’ LIVMARLI (maralixibat oral solution) has been authorized by Health Canada for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS).
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 Chief Executive Officer at Mirum. “We are grateful to the patients, families, clinicians and ALGS community who made this approval possible.”
“There have been meaningful advances in the treatment of Alagille syndrome with the approval of LIVMARLI and I am pleased that it is now available in Canada where I have seen, firsthand, the critical need for an approved therapy to address the itch caused by cholestasis. The years of data collected have shown that LIVMARLI can provide tremendous benefit, potentially giving many patients the opportunity to live transplant-free,” said Dr. Binita Kamath, Hospital for Sick Children (SickKids), 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.”
“We are thrilled that patients in Canada will now have access to LIVMARLI as we have seen the powerful impact it can have on people living with life-altering pruritus,” said Roberta Smith, president of the Alagille Syndrome Alliance. “I know from my own experience how painful it is to watch your child suffer and how disruptive this disease can be for the whole family. It is wonderful to know that patients in Canada can have a chance at a life less burdened by cholestatic 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.
In addition to this 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 U.S., Canada and Europe are being pursued by partners and distributors in key markets globally.