Altuviiio approved to treat hemophilia A in Canada
Once-a-week infusions aimed at preventing, controlling bleeds
Health Canada has approved Altuviiio (efanesoctocog alfa) to treat children, adolescents, and adults with hemophilia A, with the aim of preventing and controlling bleeds, including those occurring around the time of surgery.
“This approval represents important progress in the treatment of patients with hemophilia A in Canada,” Stephanie Veyrun-Manetti, general manager of specialty care and country lead at Sanofi Canada, said in a company press release. “As we continue to push the boundaries of what’s possible, we are working towards a future where the impact of hemophilia A on daily life is minimal and patients can live without limitations.”
Sanofi, which will market the therapy in Canada, developed Altuviiio alongside Sobi.
The Canadian Hemophilia Society welcomed the decision. “The convenience of once-a-week [into-the-vein] infusions represents an important advancement, and having another treatment option available strengthens care possibilities for people living with hemophilia A,” said Emil Wijnker, the society’s president.
Hemophilia A is caused by a deficiency in a clotting protein, factor VIII (FVIII), putting patients at a higher risk of experiencing excessive and prolonged bleeding episodes. A replacement therapy, Altuviiio works to deliver a recombinant, or lab-made, version of this protein to patients. The version of FVIII in Altuviiio is modified in a way that makes it more stable and long-lasting in the body, allowing for the therapy to be given less often than standard FVIII replacement therapies when used as a routine, preventive treatment.
Bridging the limitations gap
“The ultimate treatment goal in hemophilia care is to bridge the gap between living with hemophilia and living without limitations,” said Davide Matino, MD, a hematologist at Juravinski Hospital. “With the approval of innovative therapies like Altuviiio, patients have the possibility of sustained factor levels which is setting a new standard of care that allow patients to experience extended periods of protection.”
Anthony Chan, a pediatric hematologist at Hamilton Health Sciences, said the approval of Altuviiio “underscores why the pursuit of advanced treatments is crucial for patients.”
“Hemophilia A has been seen as a condition that dictates the terms of one’s life,” Chan said. “By maintaining factor levels in the normal to near-normal range for most of the time, this is a stepping stone towards a future where hemophilia A is no longer a life-limiting condition.”
Health Canada’s approval was supported mainly by data from two Phase 3 clinical trials: XTEND-1 (NCT04161495), which tested the therapy in patients ages 12 and older, and XTEND-Kids (NCT04759131), which tested it in children younger than 12.
Data from both studies showed that prophylactic treatment with Altuviiio effectively prevented bleeds, with about two-thirds of patients in the two studies being free from bleeds over a year on the therapy.
Altuviiio is also approved to treat hemophilia A in the U.S., Japan and Taiwan, as well as in the European Union and the U.K., where it is being marketed by Sobi under the brand name Altuvoct.
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