Health Canada approves Novartis’ Fabhalta as first oral treatment for PNH

CANADA – Health Canada has approved Novartis Canada’s Fabhalta (iptacopan capsules), making it the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH) and hemolytic anemia.

This decision was based on strong clinical trial data demonstrating the therapy’s effectiveness.

The approval was supported by results from two key Phase III trials. The APPLY-PNH study focused on patients who continued to experience anemia despite previous anti-C5 treatment and found that switching to Fabhalta led to significant hemoglobin improvement without the need for red blood cell transfusions.

Additionally, the APPOINT-PNH trial included patients who had never used complement inhibitors and further confirmed the therapy’s benefits.

Fabhalta is the only Factor B inhibitor currently approved in Canada. It provides comprehensive protection against red blood cell destruction, both inside and outside blood vessels.

However, its cost-effectiveness and clinical safety are still under review by Canada’s Drug Agency and the Institut National d’Excellence en Santé et Services Sociaux.

Novartis Canada’s country president, Mark Vineis, expressed excitement about the approval, stating: “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.”

Access to Fabhalta is restricted through a controlled distribution program. Prescribers must enrol patients in the program and ensure they are vaccinated against encapsulated bacteria before starting treatment.

PNH is a rare blood disorder caused by excessive activation of the complement system, leading to the destruction of red blood cells.

Novartis Pharmaceuticals Canada, which has a team of 600 professionals, invests over US $30 million annually in research and development to address the evolving needs of patients and the healthcare system.