Health Canada Approves Bylvay™ (odevixibat) for the treatment of cholestatic pruritus in patients with Alagille Syndrome

Bylvay™ is the first and only medication approved in Canada for the treatment of both Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis  

TORONTO, Sept. 9, 2025 /PRNewswire/ -- Medison and Ipsen announced today that Health Canada has approved Bylvay™ (odevixibat) for the treatment of cholestatic pruritus in patients 12 months and older with Alagille Syndrome (ALGS). Bylvay was first approved in Canada in 2023 for the treatment of pruritus in patients aged 6 months or older with Progressive Familial Intrahepatic Cholestasis (PFIC), a progressive and life-threatening liver disease.

ALGS is a rare genetic disorder that can affect multiple organ systems, including the liver, heart, skeleton, eyes and kidneys.ⁱ It is estimated that ALGS is present in 1 in every 30,000 live births.ⁱⁱ Most of those living with ALGS experience cholestatic pruritus which can significantly impair sleep and quality of life.

"This approval marks another important milestone for Canadians impacted by Alagille syndrome, many of whom endure relentless itching and disrupted sleep," said Roberta Smith, President, Alagille Syndrome Alliance. "Having a novel treatment option provides much-needed hope and relief to our community."

Health Canada's approval of Bylvay for ALGS was supported by data from the global Phase III ASSERT study, which demonstrated the efficacy of Bylvay in reducing cholestatic pruritus associated with ALGS within weeks of treatment. ASSERT is the world's first and only Phase III trial completed in patients with ALGS.ⁱⁱⁱ The study also revealed secondary benefits, including improvements in sleep quality and reductions in bile acid levels, with a favorable safety profile.

"The approval of Bylvay in Canada is an important step forward in the treatment of Alagille syndrome," said Dr. Susan Gilmour, Professor of Pediatrics, 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."

"We are very proud to bring Bylvay to Canada as an innovative treatment option for both ALGS and PFIC," said Pamela Minden, Country Manager, Medison Canada. "This approval reflects our dedication to ensuring timely access to innovative therapies and supporting rare disease communities across Canada."

Medison and Ipsen are part of a multiregional partnership in both Canada and Israel to bring Bylvay to patients suffering from these rare diseases and their families.

About Bylvay (odevixibat)

Bylvay is a once-daily non-systemic ileal bile acid transport inhibitor (IBATi). Bylvay works in the small intestine and has minimal systemic exposure. It helps reduce itching and lower bile acid levels, two key challenges often faced by ALGS patients.

Bylvay allows for flexible dosing based on body weight. Bylvay is available as capsules which can be swallowed whole with a glass of water or opened and sprinkled on soft food or in a liquid.

Bylvay was first launched as a treatment option for patients with PFIC in the U.S. in 2021. Bylvay also received regulatory approval in the E.U. in 2021 for the treatment of PFIC in patients aged six months or older.

In June 2023, Bylvay was also approved in the U.S. for the treatment of cholestatic pruritus in patients from 12 months of age with Alagille syndrome.  Odevixibat received regulatory approval in the E.U. for treating cholestatic pruritus in Alagille syndrome in patients aged six months and older in September 2024 and is marketed under the brand name Kayfanda®. 

Please consult the Bylvay Product Monograph at https://www.medisonpharma.com/product-monographs/ for important information relating to adverse reactions, drug interactions and dosing information. The Product Monograph is also available by calling 1-800-696-1341.

Bylvay is a trademark of Albireo, an Ipsen Company, used under licenses by Medison Pharma Canada Inc.

About ASSERT

ASSERT is a double-blind, randomized, placebo-controlled trial designed to evaluate the safety and efficacy of 120 µg /kg/day odevixibat for 24 weeks in relieving pruritus in patients with ALGS with 32 sites across North America, Europe, Middle East, and Asia Pacific. The trial enrolled patients aged 0 to 17 years of age with a genetically confirmed diagnosis of ALGS.

In the primary analysis, the study met the 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 the placebo arm (p=0.002).ⁱⁱⁱ

The study also met the key secondary endpoint showing a highly statistically significant reduction in serum bile acid concentration from baseline to the average of weeks 20 and 24 (compared to the placebo arm p=0.001).ⁱⁱⁱ 

Odevixibat treatment led to improvements in multiple sleep parameters, including the percentage of days with help falling asleep and the daytime tiredness score.ⁱⁱⁱ

Odevixibat had an overall adverse event incidence similar to placebo.ⁱⁱⁱ

About Medison

At Medison, we are addressing one of the pharma industry's most pressing challenges: enabling access to breakthrough therapies for patients with severe and rare diseases in international markets where traditional commercialization models fall short. 

As the creator and leader of the global partnership category, we have built a first-of-its-kind unified commercialization platform that enables biotechs to effectively plan, optimize and launch their therapies on a global scale. 

With a strong leadership team and a proven track record, we are the partner of choice for innovative biotech companies seeking to expand beyond the US, Western Europe and Japan into regions where most of the world's population resides. Our affiliate-like model integrates commercial, medical and operational capabilities across diverse geographies, accelerating access and delivering life-changing treatments to more patients, faster. 

Medison is on a mission to ensure that every patient, regardless of where they live, has the opportunity to benefit from breakthrough therapies. For more information, please visit https://www.medisonpharma.com/

About Ipsen

We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience.

Our pipeline is fueled by external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 80 countries.

Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com.

For more information, please contact:

Medison Communications at corpcomms@medisonpharma.com

ⁱ National Organization for Rare Disorders. Alagille Syndrome. https://rarediseases.org/rare-diseases/alagille-syndrome/   Accessed July 2025
ⁱⁱ Liver Canada. Alagille Syndrome. https://liver.ca/alagille-syndrome/#fast-facts Accessed July 2025
ⁱⁱⁱ Ovchinsky N., et al. Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT); a phase 3, double-blind, randomized, placebo-controlled trial. The Lancet Gastroenterology & Hepatology, Volume 9, Issue 7, 632 – 645

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