Fabhalta now available in Canada as treatment for adults with PNH
Approved therapy indicated for patients with hemolytic anemia
Fabhalta (iptacopan) is now available in Canada, according to its developer Novartis, which noted that it’s the first — and so far, the only — approved oral treatment that can be used on its own for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia, or low red blood cell counts driven by the destruction of red blood cells.
The therapy is effective regardless of whether patients have been treated for the condition before or not.
Following Fabhalta’s approval by Health Canada earlier this year, health experts in the country reviewed how well the therapy works, how safe it is, and how it compares in value to its cost. Now those experts have recommended that Fabhalta be covered for adults with PNH who haven’t responded well to a C5 inhibitor, or who can’t tolerate such treatment, according to a company press release.
“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, president of Novartis Canada.
Novartis already markets Fabhalta in the U.S., where it was approved in 2023 for the same indication. The company said it is now working with provinces across Canada to help ensure eligible PNH patients can access the therapy. A support program called Fabhalta Assist is now in place to help patients navigate the insurance process.
Barry Katsof, president of the nonprofit Canadian Association of PNH Patients, was diagnosed with the rare acquired disease more than 20 years ago, at a time when there were no treatments available.
Now, “it is exciting to experience how the treatment landscape is evolving and improving for the PNH community,” Katsof said.
Fabhalta approval as PNH treatment based on 2 Phase 3 trials
In humans, the complement system is a group of proteins that work with the immune system to help protect the body from infections. In PNH, the complement system becomes overactive, and instead of being protective, it destroys healthy red blood cells in the body, leading to symptoms that may include anemia, fatigue, and shortness of breath.
Unlike other approved treatments that target a complement protein called C5 to block complement activation, Fabhalta is designed to inhibit complement factor B. By inhibiting this other complement protein at an early point in the cascade of complement activation, Fabhalta is expected to prevent the destruction of red blood cells occurring both inside and outside blood vessels.
Even with a C5 inhibitor, many patients still need regular blood transfusions due to persistent anemia. Fabhalta, which comes as oral capsules, offers a treatment option that may reduce or even eliminate the need for transfusions.
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.
In both the U.S. and Canada, the approval of Fabhalta was based on two Phase 3 clinical trials: APPLY-PNH (NCT04558918) and APPOINT-PNH (NCT04820530). Their results showed the therapy was able to increase the levels of oxygen-carrying hemoglobin in adults with PNH, whether they had been treated with a C5 inhibitor or not. The use of Fabhalta also reduced or eliminated the need for transfusions.
“In clinical trials, [Fabhalta] 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 for transfusions in complement inhibitor-naive patients,” said Christopher Patriquin, MD, an assistant professor of medicine and clinician investigator at the University of Toronto in Canada.
According to Patriquin, for doctors, having access to Fabhalta could mean “more flexibility in personalizing care for patients.”
Because Fabhalta may increase the risk of serious infections caused by encapsulated bacteria, its label comes with a boxed warning noting the treatment is only available in Canada through a special distribution program. Doctors must enroll patients in the program and make sure they’ve been vaccinated against encapsulated bacteria before starting treatment.
Vineis said the company believes this therapy will be of benefit for people with PNH.
“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,” Vineis said.
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