Action

The U.S. Food and Drug Administration has approved Alhemo (concizumab-mtci) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors.

Alhemo is injected subcutaneously (under the skin) daily. Recommended dosing is available in the prescribing information. 

Disease or Condition

Hemophilia A and hemophilia B are rare, inherited bleeding disorders in which the blood does not clot properly because it does not have enough blood-clotting proteins (clotting factors). Patients with hemophilia may bleed for a longer time than normal after injury or surgery. They may also have spontaneous bleeding in muscles, joints and organs, which can be life-threatening.

Traditionally, hemophilia is treated through replacement of the missing clotting factor. However, over time, people with hemophilia may develop inhibitors (antibodies) which block the activity of factor VIII or IX (clotting factors). Inhibitors can make it difficult to stop excessive bleeding and can lead to reduced efficacy of factor replacement treatment or therapy. 

It’s estimated as many as 33,000 males in the U.S. are living with hemophilia. Hemophilia A is more common (80% to 85% of the total hemophilia population) than hemophilia B. Approximately 30% of patients with hemophilia A and 5-15% of patients with hemophilia B develop inhibitors. 

Effectiveness

The efficacy and safety of Alhemo were evaluated in a multi-national, multi-center, open-label, phase 3 trial (NCT04083781) with 91 adult and 42 adolescent male patients with hemophilia A or B with inhibitors who have been prescribed, or are in need of, treatment with therapies that bypass the inhibitor effect.

Efficacy was evaluated by comparing the number of treated bleeding episodes between the Alhemo treatment group and no prophylaxis group. A ratio of the annualized bleeding rates (ABR) was estimated to 0.14, corresponding to a reduction in ABR of 86% for the Alhemo treatment group compared to the no prophylaxis group. 

Safety Information

The most common adverse reactions (reported in at least 5% of patients) to Alhemo were injection site reactions and hives (urticaria).

Hypersensitivity reactions including redness of skin (erythema), rash, itching (pruritus), and abdominal pain have occurred in patients treated with Alhemo. Patients with a history of serious hypersensitivity to Alhemo, its components, or its inactive ingredients should not use Alhemo.

Treatment with Alhemo may increase the risk of blood clots.

Designations

Alhemo received Priority Review, Breakthrough Therapy and Orphan Drug designations for this indication.