— Fabhalta is the first oral monotherapy for previously treated and treatment-naïve adults with paroxysmal nocturnal hemoglobinuria (PNH),1,2a chronic, rare and potentially life-threatening blood disorder3,4
— Now available in Canada, this new treatment option helps address significant unmet needs for adult patients with PNH; a large proportion of patients can remain anemic and dependent on blood transfusions3,4
— Canada's Drug Agency (CDA) and Institut National d'Excellence en Santé et Services Sociaux (INESSS) have recommended reimbursement of Fabhalta in the treatment of adult patients with PNH who have an inadequate response to, or are intolerant of, a C5 inhibitor5,6
— In clinical trials, treatment with Fabhalta offered superior hemoglobin improvement in the absence of red blood cell (RBC) transfusions1,7,8,9,10
Novartis Canada is pleased to announce that Fabhalta® (iptacopan capsules) is now available across Canada for adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia.1The first and only oral monotherapy treatment indicated for PNH, Fabhalta received Health Canada approval in January 2025.
“I received my PNH diagnosis over 20 years ago, when knowledge of the disorder was limited and there were no treatments available. It is exciting to experience how the treatment landscape is evolving and improving for the PNH community,” said Barry Katsof, President, Canadian Association of PNH Patients. “The availability of a new oral treatment option will provide meaningful benefits for Canadians who continue to struggle with anemia and dependence on blood transfusions, despite their current therapies.”
Complement Component C5 is a gene that encodes a component of the complement system, a part of the body's immune system that helps defend the body against injury and foreign invaders, like bacteria and viruses.11,12Existing C5 inhibitor treatments, administered as infusions, may leave PNH symptoms uncontrolled.3,4 In fact, patients on anti-C5 treatment have reported persistent anemia, with some patients requiring blood transfusions at least once per year.3,4 Fabhalta is the only Factor B inhibitor – a small-molecule therapy that treats diseases caused by dysregulation of the alternative complement pathway – providing comprehensive control of RBC destruction within and outside the blood vessels (intra- and extravascular hemolysis [IVH and EVH]) approved in Canada.
“In clinical trials, iptacopan was superior to C5 inhibition in terms of increasing hemoglobin and achievement of transfusion-avoidance in treatment-experienced patients. It was also shown to be effective in improving hemoglobin responses without the need fortransfusions in complement inhibitor-naive patients. In both studies, LDH values were well-controlled, suggesting inhibition of terminal complement function,” 7,13said Dr. Christopher Patriquin, Assistant Professor of Medicine and Clinician Investigator, University of Toronto. “The availability of a safe and effectiveoral treatment for PNH7,8,13,14offers physicians more flexibility in personalizingcare for patients, potentially easing limitations faced by those receiving injectable therapies.”
“We remain committed to innovation that improves the lives of Canadians with PNH and are proud to bring a new oral medicine to those living with this chronic and life-altering blood disease,” said Mark Vineis, Country President, Novartis Canada. “As the first and only oral treatment for PNH, we are confident the availability of this new treatment option will support improved outcomes while providing renewed hope and freedom for patients, their loved ones and the healthcare providers who care for them.”
Novartis is proud to offer a Patient Support Program (PSP), Fabhalta Assist.
The clinical effectiveness, safety and cost-effectiveness of Fabhalta in PNH has been reviewedby Canada's Drug Agency (CDA) and Institut National d'Excellence en Santé et Services Sociaux (INESSS). Both have issued a positive recommendationin favor of the reimbursement of Fabhalta in the treatment of adult patients with PNH who have an inadequate response to, or are intolerant of, a C5 inhibitor.5,6 Novartis looks forward to working with provinces so that eligible PNH patients can access this new option.
About paroxysmal nocturnal hemoglobinuria (PNH) PNH is a rare, chronic and potentially life-threatening complement-mediated blood disorder2 (a disease that occurs when the complement system is activated inappropriately or in excess).People with PNH have an acquired mutation in some of their hematopoietic stem cells (which are located in the bone marrow and can grow and develop into RBCs, white blood cells and platelets) that causes them to produce RBCs that are susceptible to premature destruction by the complement system.2,4 This leads to intravascular hemolysis (destruction of RBCs within blood vessels) and extravascular hemolysis (destruction of RBCs mostly in the spleen and liver), which cause anemia (low levels of circulating RBCs), thrombosis (formation of blood clots) and other debilitating symptomsin varying combinations and levels of severity.2-4
It is estimated that approximately 10-20 people per million worldwide live with PNH.2 Although PNH can develop at any age, it is often diagnosed in people between 30-40 years old.15,16
About Fabhalta (iptacopan capsules) Fabhalta is an oral, Factor B inhibitor of the alternative complement pathway.17,18,19Fabhalta is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia.
Fabhalta is only available through a controlled distribution program under which prescribers must enroll patients and confirm vaccination against encapsulated bacteria.1
Health Canada approval was based on the Phase III APPLY-PNH trial in patients with residual anemia despite prior anti-C5 treatment who switched to Fabhalta1,7 and supported by the Phase III APPOINT-PNH study in complement inhibitor-naïve patients.1,8
About Novartis Novartis is a focused innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach more than 250 million people worldwide.
In Canada,Novartis Pharmaceuticals Canada Inc. employs approximately 600 people to serve the evolving needs of patients and the healthcare system and invests over $30 million in R&D yearly in the country. For more information visitwww.novartis.ca.
References
SOURCE Novartis Pharmaceuticals Canada Inc.
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