Ipsen SA

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 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 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 [email protected] i National Organization for Rare Disorders. Alagille Syndrome. Accessed July 2025 ii Liver Canada. Alagille Syndrome. 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 Logo - SOURCE Medison Pharma WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Zydus plans to share full data from the trial at an upcoming scientific congress.\n Zydus Therapeutics has racked up a phase 2b/3 win in primary biliary cholangitis (PBC), clearing the path to a filing to establish the company as a challenger to Gilead Sciences, Intercept Pharmaceuticals and Ipsen.The phase 3 part of the trial randomized 149 people to receive the PPAR agonist saroglitazar or placebo. After 52 weeks of daily oral dosing, 48.5% of patients on the Zydus drug met the biochemical response, achieving the primary endpoint of the trial. Zydus plans to discuss the data with the FDA with hopes of filing for approval in the first quarter of 2026.If approved, saroglitazar will enter a market served by other drugs, including rival PPAR agonists. The FDA approved two PPAR agonists in PBC last year, clearing Gilead’s Livdelzi and Ipsen’s Iqirvo to compete for the market with Intercept’s FXR agonist Ocaliva.Gilead reported (PDF) a 62% biochemical response rate in the study that supported approval of Livdelzi. Ipsen’s Iqirvo achieved (PDF) a 51% biochemical response rate in its pivotal trial. Unlike the PPAR agonists, Intercept\'s Ocaliva carries a boxed warning. The FDA rejected a filing for full approval of the product in November—while leaving the accelerated nod in place—and flagged liver injury reports in December. When Zydus completed enrollment in its phase 3 trial last year, the lead principal investigator reported (PDF) “growing optimism” that saroglitazar can improve on the efficacy, safety and tolerability of existing treatments. The investigator was speaking before the approvals of Livdelzi and Iqirvo.The data shared by Zydus to date preclude cross-trial comparisons to assess whether saroglitazar has any potential advantages over the incumbents. The lack of a placebo response rates in Zydus’ release limits efficacy comparisons, though the absolute response rate of saroglitazar falls short of its rivals. The biotech\'s release also lacks data on itching and fatigue, symptoms the investigator named as areas in which Zydus may have an edge.  Zydus plans to share full data from the trial at a future scientific congress.

Zydus Therapeutics is planning to file for FDA approval of its liver disease candidate early next year after saroglitazar met the primary endpoint of the EPICS-III Phase IIb/III trial in patients with primary biliary cholangitis (PBC) — setting it up to compete with Gilead Sciences and Ipsen. Zydus is the US-based speciality arm of Indian pharma Zydus Lifesciences. Similar to Gilead's Livdelzi (seladelpar) and Ipsen's Iqirvo (elafibranor), saroglitazar is a PPAR agonist, but while the former is directed against the PPAR delta receptor and the latter agonises PPAR alpha and delta, Zydus' candidate targets the alpha and gamma receptors. Both saroglitazar's potential competitors were approved last summer, offering new treatments to a disease space that previously had only one option, and one plagued with safety concerns at that — Alphasigma's Ocaliva (obeticholic acid).For more insight on how physicians are responding to Livdelzi and Iqirvo's launches, see Physician Views Results: Livdelzi emerges as favourite among rivals, but headwinds haunt its path and Physician Views Results: Gilead's fashionably late arrival for Livdelzi in PBC has doctors flocking.Besting placeboThe Phase III portion of EPICS-III randomised 149 patients with PBC who had an inadequate response or intolerance to standard-of-care ursodeoxycholic acid to receive placebo or a 1-mg oral dose of saroglitazar once daily. Zydus' candidate met its primary composite endpoint, with a statistically significant treatment difference in achieving a biochemical response of 48.5% that favoured saroglitazar over placebo at week 52. The study defined biochemical response as alkaline phosphatase <1.67x upper limit of normal and with a ≥15% decrease relative to baseline; total bilirubin also needed to be ≤ upper limit of normal. The enzyme and bilirubin, a yellow pigment, are predictors of PBC disease progression. Saroglitazar also met a key secondary goal, which measured the proportion of participants with complete normalisation of alkaline phosphatase. According to Zydus, the PPAR agonist was well tolerated, with adverse events "generally balanced" between it and the placebo group. "The EPICS-III results reinforce our commitment to advancing novel treatments for chronic liver disease — an area of high unmet medical need and growing global impact," commented Zydus Lifesciences Chairman Pankaj Patel. "Saroglitazar is the first PPAR alpha/gamma agonist to demonstrate positive Phase III data in patients with PBC and has the potential to bring real value to both patients and their healthcare providers who may need more options."The company plans to file a marketing application for saroglitazar — which has fast track designation from the FDA — in the first quarter of 2026.