Concizumab approved in Canada for hemophilia B with inhibitors
The once-a-day therapy will be marketed under the brand name Alhemo
Concizumab has been approved as a preventive treatment in Canada for hemophilia B patients ages 12 and older who are positive for factor IX inhibitors and need a routine prophylactic to prevent bleeds or reduce their frequency.
This is the first approval for the daily under-the-skin injectable therapy, which will now be marketed by Novo Nordisk under the brand name Alhemo. Hemophilia B affects about 600 Canadians, according to the company.
“We are pleased to have the approval of Alhemo to expand our portfolio of innovative options for Canadians living with hemophilia B with inhibitors,” John Burrows, vice president of rare disease at Novo Nordisk Canada, said in a company press release.
In hemophilia B, genetic mutations impair the production or function of factor IX, an important blood clotting protein. Replacement therapy is a standard form of treatment that involves administering a version of this protein.
In some patients, however, the immune system can mistake the delivered clotting factor for an infectious threat and produces antibodies against the therapy. These are often referred to as inhibitors, since they can stop the medication from being effective.
“Hemophilia B is a rare disorder and those with an inhibitor to standard therapy have limited options,” Burrows said. “Living with hemophilia B can be an ongoing struggle as a result of the inability to manage uncontrolled bleeding.”
Alhemo blocks the activity of tissue factor pathway inhibitor (TFPI), a protein that normally prevents blood clotting. Blocking the clot-stopping protein is expected to promote blood clotting and prevent bleeds.
“The approval of treatments like Alhemo will provide benefits to both patients and caregivers. This new treatment option will allow them to take control of their bleeding episodes effectively, improving their quality of life,” Burrows said.
Novo Nordisk sponsored a Phase 3 clinical trial called explorer7 (NCT04083781) that tested Alhemo in hemophilia A and B patients with inhibitors. The trial was paused in 2020 due to safety concerns raised by nonfatal clotting events, but was restarted later that year with additional safety monitoring precautions.
Results announced last year showed that after six months of treatment, the mean estimated bleed rate was less than two a year for patients wtih Alhemo compared to nearly 12 a year for those without prophylactic therapy — a difference of more than 85%.
Alhemo is administered by subcutaneous injection once a day at a dosage based on body weight.