Novartis Canada has received a Notice of Compliance from Health Canada for Fabhalta (iptacopan capsules) to treat adult patients with paroxysmal nocturnal haemoglobinuria (PNH).
Fabhalta is the first oral monotherapy for previously treated and treatment-naïve adults with PNH and haemolytic anaemia.
Complement Component C5 is a gene that encodes a key protein in the immune system’s complement pathway, which defends against pathogens, inflammation, and supports homeostasis.
Current C5 inhibitor treatments, given as infusions, may not fully control PNH symptoms.
Patients on anti-C5 therapies often experience persistent anaemia, with some needing blood transfusions annually.
Fabhalta is the only Factor B inhibitor approved in Canada.
This small-molecule therapy targets the alternative complement pathway, offering comprehensive control of RBC destruction both inside and outside blood vessels.
Novartis Canada country president Mark Vineis said: “We are proud that Fabhalta has received approval to bring a new oral medicine to Canadians living with PNH, a chronic and life-altering blood disease.
“This new treatment option provides renewed hope for patients, their loved ones and the healthcare providers who care for them.”
Health Canada approved Fabhalta based on the Phase 3 APPLY-PNH trial, which evaluated patients with residual anaemia despite prior anti-C5 treatment, who switched to the Novartis’ drug.
The approval is further supported by the Phase 3 APPOINT-PNH study, which focused on complement inhibitor-naïve patients.
In clinical trials, Fabhalta showed superior haemoglobin improvement without the need for red blood cell (RBC) transfusions.
The clinical effectiveness, safety, and cost-effectiveness of the drug are currently under review by Canada’s Drug Agency (CDA) and the Institut National d’Excellence en Santé et Services Sociaux (INESSS).
Iptacopan is available only through a controlled distribution programme. Prescribers must enrol patients and confirm vaccination against encapsulated bacteria.
In December 2024, the Swiss drugmaker announced positive topline results for Fabhalta from Phase 3B APPULSE-PNH clinical study.
The drug has also shown sustained, clinically meaningful results in Phase 3 APPEAR-C3G study of patients with C3 glomerulopathy (C3G). These results were announced in October 2024.
