Health Canada Greenlights Novartis’ Fabhalta: A Breakthrough Treatment for PNH Patients

Health Canada has given the green light to Novartis Canada’s Fabhalta (iptacopan capsules). This is significant because it is the first oral monotherapy available for adults suffering from paroxysmal nocturnal haemoglobinuria (PNH) and haemolytic anemia.

Fabhalta’s approval comes after the Phase III APPLY-PNH trial, which examined patients who still had anemia after other treatments. Participants switched to Fabhalta, showing impressive results.

Another supportive study, the Phase III APPOINT-PNH trial, involved patients who had not received any prior complement inhibitor treatments. Both trials confirmed that Fabhalta improves hemoglobin levels without requiring blood transfusions.

As the only Factor B inhibitor approved in Canada, Fabhalta plays a crucial role in managing red blood cell destruction inside and outside blood vessels, making it a unique treatment option.

Health authorities in Canada are currently reviewing its safety and value for money.

Mark Vineis, the country president for Novartis Canada, expressed pride in being able to offer this new oral medication to Canadians affected by PNH. He noted that Fabhalta offers hope to both patients and their healthcare providers.

However, access to Fabhalta is controlled. Doctors must enroll patients and confirm their vaccinations against specific bacteria before they can prescribe it.

PNH is a rare blood disorder caused by the overactivation of the complement system, affecting patients’ quality of life. Novartis Canada is dedicated to meeting the needs of patients with a robust team and significant investments in research and development.

Every year, the company invests over $30 million in R&D to improve treatments and outcomes for patients in Canada.