Bylvay now approved in Canada to treat adults, children with Alagille
Oral medication known to ease severe itching in liver disease patients
Bylvay (odevixibat), an oral medication used to treat cholestatic pruritus — severe itching that results from a buildup of bile acids used in digestion — is now approved in Canada for people with Alagille syndrome.
The decision by Health Canada will make the Ipsen medication available to adults and children, ages 1 and older, with Alagille.
Bylvay already was available in Canada, where it is marketed by Medison Pharma, for treating pruritus in patients 6 months and older with another rare liver disease called progressive familial intrahepatic cholestasis (PFIC).
“We are very proud to bring Bylvay to Canada as an innovative treatment option for both [Alagille syndrome] and PFIC,” Pamela Minden, country manager at Medison Canada, said in a company press release announcing the therapy’s approval.
With that decision, Bylvay became the first and only medication approved in Canada to treat both Alagille syndrome and PFIC. For Minden, this “reflects our dedication to ensuring timely access to innovative therapies and supporting rare disease communities across Canada.”
Alagille syndrome and PFIC are marked by cholestasis, or slowed bile flow from the liver. Bile, produced in the liver, is normally transported to the intestine, where it helps with digestion. Slowed flow causes bile acids — the main component of bile — to build up in the liver and leak into the blood, leading to symptoms such as cholestatic pruritus.
Bylvay designed to reduce bile acid buildup, easing itching
Bylvay works to block the action of the ileal bile acid transporter (IBAT), a protein that reabsorbs bile acids from the intestine back into the liver through the blood. By blocking IBAT, Bylvay helps bile acids exit through the intestine, reducing their buildup in the blood and liver, and easing cholestatic pruritus.
Available as a once-daily capsule, Bylvay is approved in the U.S. to treat pruritus caused by Alagille syndrome — also in patients 12 months and older — and PFIC, in individuals ages 3 months and older. In the European Union, the therapy is approved as Bylvay for treating PFIC and as Kayfanda for Alagille syndrome in patients 6 months and older.
This approval offers physicians an additional tool to help address the often-debilitating symptoms of [Alagille syndrome], which can pose immense challenges for both patients and caregivers.
“The approval of Bylvay in Canada is an important step forward in the treatment of Alagille syndrome,” said Susan Gilmour, MD, a professor of pediatrics and director of the pediatric liver transplant program at the University of Alberta in Canada. “This approval offers physicians an additional tool to help address the often-debilitating symptoms of this condition, which can pose immense challenges for both patients and caregivers.”
As in the U.S. and the European Union, the approval of Bylvay in Canada drew on data from ASSERT (NCT04674761), a Phase 3 clinical study involving 52 children and adolescents with Alagille syndrome. Over six months, Bylvay was shown to reduce the frequency of scratching due to cholestatic pruritus versus a placebo.
Bylvay met the study’s main goal of significantly easing pruritus as measured by the PRUCISION Observer-Reported Outcome scratching scale, after 24 weeks of treatment, compared with the placebo. This scale rates scratching from 0 to 4, with higher scores indicating more scratching.
Treatment with Bylvay also resulted in significantly lower levels of circulating bile acids and better sleep, including the percentage of days patients needed help to fall asleep and the degree of tiredness during the day. The most common side effects reported with Bylvay were vomiting, diarrhea, abdominal pain, and weight loss.
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