Funding for medicines that help the local boy cope with ALGS is encouraged

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The mother of a boy born with a debilitating liver disease that causes uncontrollable itching is hoping the medication that’s helping her son thrive will become widely available in Canada.

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Tara Jaques said her 12-year-old son Chord, born with cholestatic liver characteristic of Alagille syndrome (ALGS), has seen incredible improvement over the past two years he’s been part of a Livmarli trial.

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It’s absolutely thriving, Jaques said. He’s growing, he’s happy, he’s doing well in school, she said.

She added that Chord will start playing tennis and be part of a dance recital.

He was able to have a real, full and happy life.

Earlier this month, it was announced that the Canadian Agency for Drugs and Technologies in Health (CADTH) was recommending reimbursement to public drug plans across Canada for Livmarli, used to treat cholestatic pruritic disease in patients with Alagille (ALGS). However, the Institut national de excellence en sant et services sociaux, Quebec’s equivalent of the CADTH, has issued a negative recommendation.

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Approximately one in 30,000 children is born with ALGS.

While Chord is thriving today, it wasn’t always that way.

The first few years of agreements were a misery for him. People born with a cholestatic liver have too few or too small bile ducts, which causes the body to reabsorb bile and itch from the inside out.

Jaques said those early years when Chord was consumed by the itch were very, very hard.

The whole family is affected when a member has ALGS, she said, recalling that there was little sleep, Chord needed a feeding tube, constantly bled from scratching and couldn’t concentrate in school.

There was almost no quality of life.

He said Health Canada’s approval of Livmarli last year did not automatically provide access to the drug for cholestatic liver patients who were waiting.

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Knowing what other families are going through, Jaques would like Livmarli to be widely available in Canada as soon as possible.

It’s frustrating because there is a drug available that could help them and it’s taking so long to get to them, he said.

Livmarli is the first and only drug approved to treat cholestatic pruritus in patients with Alagille syndrome in Canada, according to a news release from California-based Mirum Pharmaceuticals Inc., which makes the drug. The drug is approved for the treatment of cholestatic pruritus in patients with (ALGS) in the US and Europe, the company said.

Accelerating access in all provinces to this meaningful treatment is critical, Mirum CEO Chris Peetz said in the release. These patients suffer greatly from the debilitating and disruptive effects of Alagille syndrome, which often begins in early childhood.

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Alagille Syndrome Alliance President Roberta Smith, whose daughter has ALGS, called the CADTH recommendation incredibly significant as it means hope for patients and their families who have suffered from disturbing and painful effects of itching for too long.

Although Chord is doing well today, Jaques said the only way to possibly reduce the impacts of ALGS is if he gets a liver transplant. There is no cure for ALGS.

They are not sure if a transplant will take place, he said. As long as your liver disease is present, you will need this medicine to control your itching.

eshreve@postmedia.com

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