Health Canada Approves Bylvay for Alagille Syndrome Treatment, Marking First Dual-Indication Rare Disease Therapy
Key Insights
Health Canada has approved Bylvay (odevixibat) for treating cholestatic pruritus (search) in patients 12 months and older with Alagille Syndrome (search), making it the first medication approved in Canada for both ALGS and PFIC.
The approval was supported by the Phase III ASSERT study, which demonstrated statistically significant improvements in pruritus scores (p=0.002) and bile acid reduction (p=0.001) compared to placebo.
Alagille Syndrome (search) affects 1 in 30,000 live births and causes debilitating itching that significantly impairs sleep and quality of life in most patients.
Medison (search) and Ipsen announced that Health Canada has approved Bylvay (odevixibat) for the treatment of cholestatic pruritus (search) in patients 12 months and older with Alagille Syndrome (search) (ALGS). This approval establishes Bylvay as the first and only medication approved in Canada for treating both Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis (search) (PFIC), following its initial Canadian approval in 2023 for PFIC patients aged 6 months or older.
Addressing Critical Unmet Need in Rare Disease
Alagille Syndrome (search) is a rare genetic disorder affecting multiple organ systems, including the liver, heart, skeleton, eyes and kidneys. The condition is estimated to occur in 1 in every 30,000 live births. Most patients with ALGS experience cholestatic pruritus (search), which can significantly impair sleep and quality of life.
"This approval marks another important milestone for Canadians impacted by Alagille syndrome (search), many of whom endure relentless itching and disrupted sleep," said Roberta Smith, President of the Alagille Syndrome Alliance. "Having a novel treatment option provides much-needed hope and relief to our community."
Phase III ASSERT Study Demonstrates Efficacy
Health Canada's approval was supported by data from the global Phase III ASSERT study, the world's first and only Phase III trial completed in patients with ALGS. The double-blind, randomized, placebo-controlled trial evaluated the safety and efficacy of 120 μg/kg/day odevixibat for 24 weeks across 32 sites in North America, Europe, Middle East, and Asia Pacific.
The study enrolled patients aged 0 to 17 years with genetically confirmed ALGS diagnosis. In the primary analysis, ASSERT met its primary endpoint, showing highly statistically significant improvement in pruritus as measured by the PRUCISION Observer-Reported Outcome scratching score (0-4 point scale) from baseline at month 6 (weeks 21 to 24), compared to placebo (p=0.002).
The trial also achieved its key secondary endpoint, demonstrating a highly statistically significant reduction in serum bile acid concentration from baseline to the average of weeks 20 and 24 compared to placebo (p=0.001). Additional benefits included improvements in multiple sleep parameters, such as the percentage of days with help falling asleep and daytime tiredness scores.
Favorable Safety Profile
Odevixibat demonstrated an overall adverse event incidence similar to placebo in the ASSERT study. The drug works as a once-daily non-systemic ileal bile acid transport inhibitor (search) (IBATi) in the small intestine with minimal systemic exposure, helping to reduce itching and lower bile acid levels.
"The approval of Bylvay in Canada is an important step forward in the treatment of Alagille syndrome (search)," said Dr. Susan Gilmour, Professor of Pediatrics and Director of Pediatric Liver Transplant at University of Alberta. "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."
Flexible Dosing and Administration
Bylvay allows for flexible dosing based on body weight and is available as capsules that can be swallowed whole with water or opened and sprinkled on soft food or in liquid. This formulation flexibility is particularly important for pediatric patients.
Global Regulatory Progress
The Canadian approval follows a series of international regulatory milestones for odevixibat. Bylvay was first launched for PFIC treatment in the U.S. in 2021 and received E.U. approval for PFIC in patients aged six months or older the same year. In June 2023, the drug was approved in the U.S. for cholestatic pruritus (search) in ALGS patients from 12 months of age. Most recently, odevixibat received E.U. regulatory approval for treating cholestatic pruritus in Alagille syndrome (search) patients aged six months and older in September 2024, where it is marketed under the brand name Kayfanda.
"We are very proud to bring Bylvay to Canada as an innovative treatment option for both ALGS and PFIC," said Pamela Minden, Country Manager of Medison (search) Canada. "This approval reflects our dedication to ensuring timely access to innovative therapies and supporting rare disease communities across Canada."