The mother of a child born with a debilitating liver disease that causes uncontrollable itching is hoping the medication that is helping her son thrive will become widely available in Canada.
The mother of a child born with a debilitating liver disease that causes uncontrollable itching is hoping the medication that is helping her son thrive will become widely available in Canada.
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Tara Jaques said her son Chord, 12, born with a cholestatic liver – the hallmark of Alagille syndrome (ALGS) – has seen incredible improvement during the last two years he has been part of a Livmarli trial.
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“He’s absolutely thriving,” Jaques said. “He’s growing, he’s happy, he’s doing well in school,” she said.
She added Chord is going to start playing tennis and be part of a dance recital.
“He’s been able to have a real, full, happy life.”
Earlier this month, it was announced the Canadian Agency for Drugs and Technologies in Health (CADTH) recommended reimbursement to public drug plans across Canada for Livmarli, used to treat cholestatic pruritus in patients with Alagille syndrome (ALGS). However, the National Institute of Excellence in Health and Social Services, the Quebec equivalent to the CADTH, has issued a negative recommendation.
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About one in every 30,000 children is born with ALGS.
Although Chord is thriving today, that wasn’t always the case.
Chord’s early years were misery for him. People born with a cholestatic liver have either too few or too small bile ducts, causing the body to reabsorb bile making it itchy from the inside out.
Jaques said those early years when Chord was “consumed by the itch were really, really hard.”
The entire family is affected when one member has ALGS, she said, recalling there was little sleep, Chord needed a feeding tube, he was constantly bleeding from scratching, and he couldn’t focus in school.
“There was just almost no quality of life.”
She said Health Canada’s approval of Livmarli last year didn’t 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 to see Livmarli become widely available in Canada as soon as possible.
“It’s frustrating, because there’s a medication available that could help them and it’s taking so long to get it to them,” she said.
Livmarli is the first and only approved medication to treat cholestatic pruritus in patients with Alagille syndrome in Canada, states a media release from California-based Mirum Pharmaceuticals Inc. which produces 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,” said Mirum CEO Chris Peetz in the release. “These patients suffer greatly from the debilitating and disruptive effects of Alagille syndrome, which often starts in early childhood.”
Alagille Syndrome Alliance president Roberta Smith, whose daughter has ALGS, called the CADTH recommendation “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.”
Although Chord is doing well today, Jaques said the only way he will be cured of ALGS is if he gets a liver transplant.
They are unsure if a transplant will happen, she said. “As long as his liver disease is present, he will need this medication to control his itch.”
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