Health Canada has authorized the use of maralixibat oral solution (Livmarli®) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS), as recently announced by Mirum Pharmaceuticals in a press release.
“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.”
Health Canada’s authorization is based on data from the pivotal ICONIC study, which includes 6 years of data from the maralixibat clinical program. The study has provided robust evidence demonstrating statistically significant and clinically meaningful reductions in pruritus vs placebo, along with significant and durable reductions in serum bile acids.
Binita Kamath, MD, Hospital for Sick Children (SickKids) in Toronto, Canada, celebrated the approval. “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.”
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“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,” Dr. Kamath added.
The results from the maralixibat studies have been featured in several publications, including The Lancet and Hepatology.
“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.”
Maralixibat has already received approval in the United States and Europe for the treatment of cholestatic pruritus in patients with ALGS.
In addition, Mirum Pharmaceuticals has submitted supplemental new drug applications for approval of maralixibat in progressive familial intrahepatic cholestasis.
Mirum’s LIVMARLI® now authorized in Canada for cholestatic pruritus in patients with Alagille syndrome. News release. Mirum Pharmaceuticals, Inc.; July 25, 2023.