Canada Approves Rebinyn as a Preventive for Children With Hem B
Rebinyn now available to all hemophilia B patients to reduce number of bleeds
Health Canada has expanded its approval of Rebinyn (nonacog beta pegol) as a routine preventive treatment for bleeding episodes in hemophilia B to patients under age 18.
The therapy, developed and marketed by Novo Nordisk, is now available to all hemophilia B patients as a preventive (prophylactic) and on-demand treatment for spontaneous bleeds, and to manage those occurring around the time of surgery.
“Novo Nordisk is pleased to expand the Rebinyn indication to ensure Canadians with rare genetic bleeding disorders have innovative treatment options to support their needs,” John Burrows, vice president of rare disease at Novo Nordisk Canada, said in a press release. “We are committed to making a meaningful difference in the rare disease community.”
Rebinyn was previously approved in Canada in 2017 as on-demand treatment and to prevent bleeds in and around the time of surgery in all hemophilia B patients, and as a routine prophylactic treatment in adults, 18 and older.
For the expanded approval, Health Canada reviewed data that included children and adolescents previously treated with Rebinyn prophylactically for up to eight years, and previously untreated patients who were on preventive therapy for up to six years.
The therapy was also approved in the U.S. in 2017 as an on-demand or surgical treatment for hemophilia B in patients of all ages. Its approval was recently expanded, allowing the therapy to be used as a routine preventive treatment for all patients.
Rebinyn is a replacement therapy designed to maintain high enough levels of factor IX (FIX) — the blood clotting protein that is missing in people with hemophilia B — and to reduce the risk of bleeds. It is an extended half-life product, meaning therapeutic FIX was modified to last longer in the body and reduce the frequency of dosing compared with other replacement therapies.
All approvals were primarily supported by data from four Phase 3 trials that were part of the PARADIGM program. These studies evaluated the treatment’s effectiveness and safety for routine preventive and on-demand treatment, as well as surgical management in more than 100 previously treated boys and men with hemophilia B. Additional long-term PARADIGM studies are ongoing.
“We will continue to deliver innovative therapies that play an important role in improving hemophilia care for patients across Canada, and demonstrate our commitment to patients, caregivers, and healthcare professionals,” Burrows added.