Health Canada approves IQIRVO® as a first-in-class dual-peroxisome proliferator-activated receptor treatment for primary biliary cholangitis Français
IQIRVO® (elafibranor) is the first new medicine approved in nearly a decade for the treatment of rare cholestatic liver disease Primary Biliary Cholangitis
MISSISSAUGA, ON, April 28, 2025 /CNW/ - Ipsen Biopharmaceuticals Canada Inc. today announced Health Canada has issued a Notice of Compliance with Conditions (NOC/c) approving IQIRVO® (elafibranor) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA.1
PBC is a chronic, rare autoimmune liver disease where the immune system attacks the liver causing slow, progressive damage to the bile ducts.2,3 Canada has one of the highest rates of PBC incidence and prevalence worldwide, affecting 318 individuals for every million Canadians (approximately 13,000 people).4,5 PBC is a lifelong condition that can worsen over time if not effectively treated, leading to liver transplant and in some cases, premature death.6
"The treatment landscape for PBC has not seen advancement in nearly a decade, making IQIRVO® a significant step forward in filling an unmet need for Canadian patients," said Dr. Mark G. Swain, Professor of Medicine, Cal Wenzel Family Foundation Chair in Hepatology. "If left untreated, PBC can progress and lead to liver failure, which is why second line options are needed. Based on the Phase III ELATIVE® trial, IQIRVO® offers the potential to significantly reduce serum ALP levels, providing patients with an effective second line treatment option."
IQIRVO® is a first-in-class oral, once-daily peroxisome proliferator-activated receptor (PPAR) agonist. The accelerated approval by Health Canada was based on data from ELATIVE® trial, a randomized, placebo-controlled, phase III study that assessed alkaline phosphatase (ALP) and bilirubin as a composite biochemical surrogate endpoint.7 In the study, IQIRVO® demonstrated a statistically significant treatment benefit with 51% of patients on IQIRVO® achieving a biochemical response compared with 4% on the placebo arm for a 47% treatment difference regardless of baseline ALP.8 Biochemical response was defined as ALP less than 1.67 Upper Limit of Normal (ULN), an ALP decrease of greater than or equal to 15% from baseline and total bilirubin (TB) ≤ ULN at week 52.9
"PBC is a rare, slowly progressive autoimmune liver disease and because it is somewhat 'invisible' it may be misunderstood and dismissed by friends, family and even the medical community," said Gail Wright, President of Canadian PBC Society. "The impact of PBC on day-to-day life can be devastating, especially for those PBC patients that suffer from severe fatigue and debilitating itch. Unrelenting symptoms affect their ability to work and carry out tasks of daily living, which can result in social isolation, anxiety and depression. The Canadian PBC Society welcomes this approval underscoring the importance of having access to well-tolerated and effective treatment options for Canadians living with PBC that potentially slow disease progression."
PBC is the most common autoimmune liver disease, primarily affecting women aged 35-60, and impacting 1 in 1,000 women over the age of 40.10 Men are also susceptible but to a much lesser degree, with a ratio of one affected man for every nine affected women.11
"For many people living with PBC, the existing treatment does not control the condition, said Ed Dybka, General Manager, Ipsen Biopharmaceuticals Canada Inc. "IQIRVO® is a first-in-class treatment and this approval underscores Ipsen's commitment to being a leader in developing innovations that address unmet needs for Canadians living with rare disease."
About the Phase III ELATIVE® Trial12
The ELATIVE® trial is a multi-centre, randomized double-blind, placebo-controlled Phase III clinical trial (n=161) that evaluated the efficacy and safety of IQIRVO® 80mg once daily plus UDCA (n=108) versus placebo plus UDCA (n=53). IQIRVO® or placebo was administered in combination with UDCA in 95% of patients and as monotherapy in 5% of patients who were unable to tolerate UDCA. The 52-week study was completed by 92% of participants with 97% of those who completed the study continuing in an extension study. The results were published in the New England Journal of Medicine.13
- The ELATIVE® trial demonstrated that IQIRVO® had a statistically significant treatment benefit with 51% of patients on IQIRVO® achieving a biochemical response compared with 4% on the placebo arm, a treatment difference of 47% (95% CI 32, 57; p<0.0001) regardless of baseline ALP. Biochemical response was defined as ALP less than 1.67 Upper Limit of Normal (ULN), an ALP decrease of greater than or equal to 15% from baseline and total bilirubin (TB) ≤ ULN at week 52.
- ALP normalization at week 52 was a key secondary endpoint with 15% of IQIRVO®-treated patients demonstrating normalization versus 0% placebo (p=0.002).
- The significant biochemical response to IQIRVO® was further supported by data demonstrating reductions from baseline in ALP levels were sustained through week 52 and response was rapid, seen as early as Week 4 in the IQIRVO® group.
- The most commonly reported (>10%) adverse drug reactions associated with IQIRVO® in more than 10% of study participants (n=108) were abdominal pain (11%), diarrhea (11%), nausea (11%) and vomiting (11%).14 These were non-serious and mild to moderate in severity. The most common adverse drug reaction leading to treatment discontinuation was blood CPK increased (3.7%).15
About IQIRVO®
IQIRVO® (pronounced EYE-KER-VO) is an oral, once-daily, peroxisome proliferator-activated receptor (PPAR) agonist indicated for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.16 While the mechanism is not well understood, pharmacological activity that is potentially relevant to IQIRVO® therapeutic effects includes inhibition of bile acid synthesis through activation of PPAR-alpha and PPAR-delta.17 IQIRVO® has been approved by the Food and Drug Administration (FDA) in the United States, the European Medicines Agency (EMA) in Europe and the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA). The Health Canada approval of IQIRVO® further strengthens Ipsen's portfolio of treatments for rare cholestatic liver diseases available to patients in Canada.
IQIRVO® is an 80 mg tablet administered orally once daily.18 To support access to IQIRVO® for eligible individuals in Canada, the IPSEN CARES® patient support program is available as a resource to people living with PBC and their caregivers to provide educational support and address coverage, access and reimbursement questions (1-855-215-2288).
IQIRVO® has been granted a Notice of Compliance with Conditions (NOC/c) and Health Canada has provided market authorization to this product on the condition that Ipsen carries out additional clinical trials to verify the anticipated benefit within an agreed upon time frame.19 The confirmatory trial for IQIRVO®, ELFIDENCE, is ongoing.
For more safety and prescribing information, please refer to the IQIRVO® product monograph.
About Ipsen in North America
Ipsen is a global biopharmaceutical company focused on innovation and specialty care. At Ipsen, we focus our resources, investments and energy on discovering, developing and commercializing medicines in three key therapeutic areas – Oncology, Rare Disease and Neuroscience. Our North American operations are located in Cambridge, Massachusetts, one of the company's three global hubs. Based in the heart of Kendall Square, our fully integrated biopharmaceutical business includes Commercial, Research & Development, Manufacturing, and Business Development. Combined with our Canadian headquarters in Mississauga, Ontario, and other locations, we employ approximately 600 people in North America. For more information, please visit www.ipsen.com/canadaEN.
ELATIVE is a registered trade-mark of Genfit, used under licence by Ipsen Biopharmaceuticals Canada Inc.
IQIRVO is a registered trade-mark of Genfit S.A., used under licence by Ipsen Biopharmaceuticals Canada Inc.
Ipsen Cares is a registered trade-mark of Ipsen S.A.
References
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2 NIH/CMAJ. Epidemiology and liver transplantation burden of primary biliary cholangitis: a retrospective cohort study: https://pmc.ncbi.nlm.nih.gov/articles/PMC6303180/#b1-cmajo.20180029 |
3 Liver Canada. Primary biliary cholangitis. https://liver.ca/primary-biliary-cholangitis-2/ |
4 Canadian PBC Society. What is PBC. https://pbc-society.ca/what-is/ |
5 NIH/CMAJ. Epidemiology and liver transplantation burden of primary biliary cholangitis: a retrospective cohort study: https://pmc.ncbi.nlm.nih.gov/articles/PMC6303180/#b1-cmajo.20180029 |
6 Ipsen. Rare cholestatic liver disease. https://www.ipsen.com/rare-cholestatic-liver-disease/ |
7 IQIRVO® Product Monograph. Ipsen Biopharmaceuticals Canada Inc. 25 April 2025 |
8 IQIRVO® Product Monograph. Ipsen Biopharmaceuticals Canada Inc. 25 April 2025 |
9 IQIRVO® Product Monograph. Ipsen Biopharmaceuticals Canada Inc. 25 April 2025 |
10 Canadian PBC Society. What is PBC. https://pbc-society.ca/what-is/ |
11 Canadian PBC Society. What is PBC. https://pbc-society.ca/what-is/ |
12 Kowdley. K.V, et al. Efficacy and Safety of Elafibranor in Primary Biliary Cholangitis. NEJM. 2023. DOI: 10.1056/NEJMoa2306185 |
13 Kowdley. K.V, et al. Efficacy and Safety of Elafibranor in Primary Biliary Cholangitis. NEJM. 2023. DOI: 10.1056/NEJMoa2306185 |
14 IQIRVO® Product Monograph. Ipsen Biopharmaceuticals Canada Inc. 25 April 2025 |
15 IQIRVO® Product Monograph. Ipsen Biopharmaceuticals Canada Inc. 25 April 2025 |
16 IQIRVO® Product Monograph. Ipsen Biopharmaceuticals Canada Inc. 25 April 2025 |
17 IQIRVO® Product Monograph. Ipsen Biopharmaceuticals Canada Inc. 25 April 2025 |
18 IQIRVO® Product Monograph. Ipsen Biopharmaceuticals Canada Inc. 25 April 2025 |
19 IQIRVO® Product Monograph. Ipsen Biopharmaceuticals Canada Inc. 25 April 2025 |
SOURCE Ipsen Biopharmaceuticals Canada Inc.
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