With Livmarli approval, PFIC patients in Canada have new itch relief option
Oral therapy OK'd for ages 1 and older with itching due to cholestasis
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Health Canada has approved Mirum Pharmaceuticals’ oral therapy Livmarli (maralixibat) for cholestatic pruritus — that is, itching due to impaired bile flow — in adults and children, ages 1 and older, with progressive familial intrahepatic cholestasis (PFIC).
The decision was welcomed by the developer, who noted that PFIC affects approximately 1 of every 50,000 to 100,000 babies born in Canada.
“Health Canada’s authorization of Livmarli for the treatment of cholestatic pruritus in patients with PFIC aged 12 months and older has the potential to be transformational for patients living with the severe burden of symptoms associated with this disease,” Jamie Twiselton, general manager at Mirum Pharmaceuticals Canada, said in a press release from the parent company.
The Canadian approval applies to an oral solution formulation of Livmarli, as well as a tablet formulation that’s indicated for patients who weigh at least 22 kg (about 48 lbs) who can swallow tablets. Having both versions available “offers meaningful flexibility and convenience to support treatment as patients with PFIC age,” Twiselton said.
Bile is a digestive fluid produced by the liver and normally delivered to the intestines via a series of tubes called bile ducts. PFIC encompasses a group of genetic disorders in which the flow of bile is slowed or stopped, leading to bile building up in the liver and leaking out into the bloodstream.
For many patients, this leads to cholestatic pruritus, meaning itching (pruritus) due to stalled bile flow; this condition is known as cholestasis.
There are several PFIC types, each caused by mutations in different genes.
Livmarli approval in Canada welcomed by advocacy group
Livmarli is a cholestasis treatment that works to reduce the amount of bile that is carried from the intestine back into the liver to be recycled. It limits how much bile builds up in the liver and leaks into the bloodstream, which can ultimately help ease cholestatic pruritus.
“PFIC is a complex condition to treat, and pruritus is often among the most burdensome symptoms for children and families,” said Susan M. Gilmour, MD, a professor at the University of Alberta. “An authorized option in Canada that addresses cholestatic pruritus and is indicated across multiple PFIC types, while offering flexibility in formulation, represents an important advancement for clinical care.”
Health Canada’s approval of Livmarli for PFIC was based mainly on data from the Phase 3 MARCH-PFIC clinical trial (NCT03905330), which tested the therapy against a placebo in 93 children and adolescents with various types of PFIC. The results showed that Livmarli was better than the placebo at easing itch, with consistent findings across all evaluated PFIC types.
For many patients, the itch is so severe it impacts sleep, nutrition and child development. … [This approval] offers hope for meaningful relief from pruritus, and the availability of both liquid and tablet formulations offers treatment flexibility for patients.
The PFIC Network, a nonprofit advocacy group, said the approval is good news for those in Canada who live with the chronic condition.
“For many patients, the itch is so severe it impacts sleep, nutrition and child development,” said Emily Ventura, the PFIC Network’s cofounder and executive director.
Ventura added that the new Canadian approval “offers hope for meaningful relief from pruritus, and the availability of both liquid and tablet formulations offers treatment flexibility for patients.”
Both the oral solution and tablet formulations of Livmarli are also approved in Canada to treat cholestatic pruritus in people with Alagille syndrome, another disorder that affects bile flow. The therapy is also authorized for PFIC and Alagille in the U.S., the European Union, and Japan.
