Health Canada has approved Novartis Canada’s Fabhalta (iptacopan capsules) as the first oral monotherapy to treat adults with paroxysmal nocturnal haemoglobinuria (PNH) and haemolytic anaemia.

The Notice of Compliance issued by Health Canada approving Fabhalta is supported by findings from the Phase III APPLY-PNH trial, which involved subjects with residual anaemia despite previous anti-C5 treatment who switched to the therapy.

Further backing comes from the Phase III APPOINT-PNH trial, which included complement inhibitor-naïve subjects.

Clinical trials have demonstrated that the therapy provides superior haemoglobin improvement without the need for red blood cell transfusions.

The therapy is the only Factor B inhibitor approved in the country, offering comprehensive control of red blood cell destruction, both within and outside blood vessels.

Its clinical safety and cost-effectiveness are currently under review by Canada's Drug Agency and Institut National d'Excellence en Santé et Services Sociaux.

Novartis Canada country president Mark Vineis stated: "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."

The therapy is accessible only via a controlled distribution programme, which requires prescribers to enrol subjects and confirm their vaccination against encapsulated bacteria.

PNH is characterised by the inappropriate or excessive activation of the complement system.

Novartis Pharmaceuticals Canada has a team of 600 focused on addressing the changing needs of patients and the healthcare system. The company invests more than $30m annually in research and development within the country.