Pruritus treatment Livmarli should be reimbursable: Canadian panel

A Canadian Agency for Drugs and Technologies in Health (CADTH) committee has recommended that Livmarli (maralixibat) be covered by the country’s publicly funded health systems as a treatment to ease itch, or pruritus, in people with Alagille syndrome.

The recommendation from the CADTH’s Canadian Drug Expert Committee brings the therapy, which last year became the first medication approved in Canada to treat pruritus caused by Alagille, a step closer to public funding.

“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,” Chris Peetz, CEO of Mirum Pharmaceuticals, which markets Livmarli, said in a company press release.

Roberta Smith, president of the Alagille Syndrome Alliance and mother of a daughter with the rare liver disease, said, “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.”

Provinces have final say on coverage

CADTH is an independent body tasked with reviewing scientific evidence to help guide decisions by policymakers and Canada’s publicly funded health insurance programs. Final decisions about coverage are determined by authorities in each Canadian province.

“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,” Peetz said.

Alagille syndrome is a genetic condition marked by problems during embryo development that lead to a wide range of symptoms, including heart and liver malformations.

Liver defects are characterized by fewer than normal bile ducts inside the liver. Bile ducts are the series of tubes that transport bile, a digestive fluid, from the liver to the small intestine. This deficit in bile ducts leads to cholestasis, or the slowing or stalling of bile flow, which results in toxic bile buildup in the liver and bile leakage into the bloodstream, causing itch.

Livmarli is designed to reduce bile buildup in the liver by blocking the activity of an enzyme that normally helps recycle bile, taking bile out of the intestines and back to the liver so it can be used again. The therapy, administered once a day as an oral solution, prompts more bile to be excreted in feces so less bile accumulates to toxic levels in the liver.

Trial data showed benefits

Previous trial data showed that Livmarli significantly eased pruritus and reduced bile acids in the bloodstream compared with a placebo in Alagille patients. Long-term Livmarli treatment was also associated with prolonged survival and a lower risk of clinically important liver outcomes, including liver transplant.

Binita Kamath, MD, senior associate scientist at The Hospital for Sick Children in Toronto and director of the hospital’s hepatology program, said the recommendation “provides a meaningful advancement in the treatment of cholestatic pruritus for patients with Alagille syndrome” and will make Livmarli “more accessible in Canada.”

Livmarli has been approved to treat cholestatic pruritus associated with Alagille syndrome in the U.S. and the European Union. The therapy was also recently approved in the U.S. to treat progressive familial intrahepatic cholestasis, a group of genetic disorders that, like Alagille syndrome, are marked by cholestasis.

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