Fabhalta approved in Canada as oral PNH treatment for adults
Therapy works to prevent destruction of red blood cells in patients
Health Canada has approved the oral therapy Fabhalta (iptacopan) as a treatment for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia.
With that decision, Fabhalta has now become the first oral medication that can be used on its own to control PNH to win approval in Canada, according to its developer Novartis.
“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,” Mark Vineis, country president of Novartis Canada, said in a company press release. “This new treatment option provides renewed hope for patients, their loved ones and the healthcare providers who care for them.”
A rare acquired disease, PNH is characterized by hemolytic anemia — low red blood cell counts driven by hemolysis, or the destruction of red blood cells — due to the abnormal activation of the complement cascade, a part of the immune system.
Fabhalta is designed to inhibit a complement protein called Factor B that normally acts earlier on in the cascade of complement activation. By targeting this earlier stage of complement activation, the therapy aims to prevent the destruction of red blood cells occurring both inside and outside blood vessels. Fabhalta has been approved in the U.S. since late 2023, and also won approval last year in the European Union.
According to Christopher Patriquin, MD, an assistant professor and clinician investigator at the University of Toronto, “a safe and effective oral option to treat PNH could be practice-changing and can relieve the treatment burden experienced by people with PNH receiving [injection or infusion] therapies.”
Health Canada decision follows approval of PNH treatment in US, EU
Fabhalta’s approval in Canada was based on data from two Phase 3 trials: APPLY-PNH (NCT04558918) and APPOINT-PNH (NCT04820530). both completed in 2023. APPLY-PNH tested Fabhalta in patients who were experiencing anemia despite receiving treatment with Soliris (eculizumab) or Ultomiris (ravulizumab), two already approved PNH medications. APPOINT-PH, meanwhile, evalauted the treatment’s use in PNH patients who hadn’t been previously given complement-inhibiting therapies.
The results from APPLY-PNH showed that Fabhalta, then still known by its chemical name, was better than the older therapies at increasing the levels of hemoglobin — the oxygen-carrying protein in red blood cells that is commonly used to assess anemia. APPOINT-PNH data consistently indicated that the therapy was effective at raising hemoglobin levels in previously untreated patients. In both trials, most patients given Fabhalta did not require blood transfusions.
“In treatment-experienced patients, [Fabhalta] was superior to [Soliris and Ultomiris] with respect to hemoglobin improvements and achievement of transfusion avoidance. It was also shown effective in treatment-naive patients, providing meaningful increases in hemoglobin without the need for transfusions,” Patriquin said.
Health Canada’s approval of [Fabhalta] now provides PNH patients options to assure that they get optimal treatment benefit.
Novartis noted that agencies from Canada’s publicly funded healthcare system are now reviewing the safety, efficacy, and cost-effectiveness of Fabhalta to guide decisions about potential coverage. In the meantime, the therapy’s approval has been welcomed by Canadian patients.
Barry Katsof, president of the Canadian Association of PNH Patients, said “Health Canada’s approval of [Fabhalta] now provides PNH patients options to assure that they get optimal treatment benefit.” He noted that, when he received his PNH diagnosis in 2003, “very few doctors were aware of PNH, let alone knowledgeable about how to treat it.”
“The availability of a new treatment was game-changing for me — my energy came back and I started to live again,” Katsof said.
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