Canada OKs Alhemo for hemophilia A patients with inhibitors

Under-the-skin injectable therapy also recently approved for hemophilia B

Steve Bryson, PhD avatar

by Steve Bryson, PhD |

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Novo Nordisk’s Alhemo (concizumab injection) has been approved in Canada for hemophilia A patients, 12 years or older, who test positive for factor VIII inhibitors.

The under-the-skin injectable therapy was recently approved by Health Canada for hemophilia B patients in the same age group with factor IX inhibitors who need a routine treatment to reduce or prevent bleeding episodes.

“We are pleased to expand the Alhemo indication to treat hemophilia A with factor VIII inhibitors,” John Burrows, vice president of rare disease at Novo Nordisk Canada, said in a company press release. “This represents significant progress for the rare disease community, and an additional option to support those living with hemophilia A with inhibitors.”

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Alhemo is antibody-based therapy option designed to promote blood clotting

The regulatory decision makes Alhemo the first approved ready-to-use under-the-skin preventive treatment for people living with hemophilia A or B with inhibitors.

Hemophilia is caused by genetic mutations that impair the production or function of proteins required for proper blood clotting. It is divided into different types, depending on the specific clotting protein that’s missing: factor VIII (FVIII) in the case of hemophilia A and factor IX in hemophilia B.

Mild hemophilia cases are characterized by prolonged bleeding after surgery or trauma. In severe cases, patients can experience prolonged bleeding from minor wounds and spontaneous bleeding into organs, joints, and muscles.

Standard replacement therapy, which can be administered on demand for acute bleeds or routinely to prevent bleeds (prophylaxis), provides a lab-made version of the missing factor to patients.

But because the delivered clotting factor comes from an external source, the immune system can produce antibodies, or inhibitors, against it, which can reduce or eliminate therapy efficacy.

Alhemo is an antibody-based therapy designed to promote blood clotting and prevent bleeding by suppressing the activity of tissue factor pathway inhibitor, a protein that prevents blood clotting under normal circumstances.

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Approvals in Canada supported by data from Phase 3 explorer7 clinical trial

The Canadian approvals were both supported by data from the Phase 3 explorer7 clinical trial (NCT04083781), which evaluated the safety and efficacy of Alhemo as a preventive treatment in 136 hemophilia A and B patients with inhibitors.

Although safety concerns related to nonfatal blood-clotting events led to a pause in the trial in 2020, it was restarted later that year with additional safety monitoring.

The first two groups of patients comprised 33 participants treated with Alhemo, administered once daily, and 19 participants who received no prophylaxis. Six-month results from these groups showed the mean estimated bleed rate dropped by more than 85%, from 12 per year without prophylactic therapy to two per year with Alhemo.

“This new indication for Alhemo builds upon Novo Nordisk’s commitment to the hemophilia community and supports our goal of improving hemophilia care for patients across Canada,” Burrows said.