Summary of Cancellation for Alhemo

This submission proposed the expansion of the indication to include patients with hemophilia A without inhibitors and hemophilia B without inhibitors.

The proposed indication was as follows:

Alhemo (concizumab injection) is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes for adolescent and adult patients (12 years of age or older) with:

• hemophilia A (congenital factor VIII [FVIII] deficiency) with or without FVIII inhibitors

• hemophilia B (congenital factor IX [FIX] deficiency) with or without FIX inhibitors.

The sponsor submitted clinical data, published studies, and labelling documentation. The pivotal clinical safety and efficacy information consisted of a Phase 3a trial (Study NN7415-4307).

Study NN7415-4307 primarily assessed the effect of concizumab prophylaxis to no prophylaxis (on-demand treatment with factor) in reducing the number of bleeding episodes in adult and adolescent patients with hemophilia A without inhibitors and hemophilia B without inhibitors.

At the time of the cancellation, the review of the submission had recently begun. Health Canada had identified some limitations in the clinical data and issued a request for clarification. The sponsor requested additional time to respond, which was granted. However, after early dialogue with Health Canada, the sponsor chose to withdraw their submission before a response was filed.

There is no expected impact for hemophilia patients using Alhemo for its currently authorized indication or who are enrolled in clinical trials.

Requests for special access to Alhemo will continue to be considered on a case-by-case basis. For more information about the Special Access Programme refer to the programme’s Web site: http://www.healthcanada.gc.ca/sap or http://www.santecanada.gc.ca/pas.