Canada approves Livmarli oral tablets for Alagille itching
New formulation aims to provide flexibility for patients
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An oral tablet formulation of Livmarli (maralixibat) is now approved in Canada to treat pruritus (itching) caused by Alagille syndrome.
An oral solution formulation of Mirum Pharmaceuticals’ therapy was previously available in the country for Alagille patients aged 1 and older. The new tablet formulation aims to offer dosing flexibility for patients weighing at least 22 kg (about 48 lbs) who can swallow tablets.
“Livmarli’s tablet formulation provides an important new option for patients with ALGS [Alagille syndrome],” Jamie Twiselton, general manager of Mirum Pharmaceuticals Canada, said in a company press release. “With a liquid formulation for younger patients and a convenient one tablet per dose option available for older patients, Livmarli now provides greater dosing flexibility, supporting continuity of care as patients with ALGS age.”
The therapy’s tablet formulation was also approved last year in the U.S. for Alagille patients weighing at least 25 kg (about 55 lbs) who are able to swallow. The original liquid formulation is cleared for use in patients 3 months and older.
Treatment aims to ease itching by slowing bile buildup
Alagille syndrome is a genetic disorder that can affect many organs throughout the body, most commonly the liver and heart. Liver involvement is typically marked by fewer-than-normal bile ducts inside the liver. Bile ducts are a series of tubes that normally carry bile, a digestive fluid, out of the liver and to the intestines.
Bile duct problems in Alagille syndrome can lead to cholestasis, in which bile flow is slowed or blocked. As a result, bile builds up to toxic levels in the liver, damaging the organ, and spills into the bloodstream, causing Alagille symptoms such as cholestatic pruritus.
“The pruritus experienced by patients with ALGS is among the most severe in any chronic liver disease and is present in most affected children by the third year of life,” Mirum said.
Livmarli is designed to block an enzyme that carries bile acids (the main component of bile) from the intestine back into the blood and liver, increasing the amount of bile acids excreted in stool. The therapy aims to reduce the bile buildup that drives itching in Alagille.
Clinical trial data showed that seven years of Livmarli treatment was associated with less itching, lower levels of liver damage markers, and better growth in nearly all children with Alagille.
“As children with Alagille syndrome grow, treatment needs can change,” said Or Steg Saban, MD, of the division of gastroenterology, hepatology, and nutrition at the University of Toronto’s Hospital for Sick Children. “The availability of a tablet formulation of Livmarli in Canada provides an additional option for appropriate patients, which may support treatment adherence and continuity of care while continuing to address cholestatic pruritus.”
