FDA Issues Complete Response Letter for Novo Nordisk’s Investigational Hemophilia Treatment

Rare Daily Staff

On April 24, 2023, Novo Nordisk received a Complete Response Letter from the U.S. Food and Drug Administration for concizumab, the company said in its first quarter 2023 financial report.

Concizumab was submitted for regulatory approval in the United States for the treatment of hemophilia A and B with inhibitors in August 2022. In the letter, the FDA requested additional information related to the monitoring and dosing of patients to ensure that concizumab is administered as intended. Furthermore, additional information on the manufacturing process was requested.

Novo Nordisk said it is evaluating the content of the CRL and will work closely with the FDA to provide the requested data. Concizumab has been approved in Canada for the treatment of hemophilia B with inhibitors and is currently under review for the treatment of hemophilia A with inhibitors.

Hemophilia is a rare, lifelong condition in which the ability of a person’s blood to clot properly is impaired, leading to excessive bleeds and spontaneous bleeds into joints that can result in joint damage and chronic pain, and potentially impact quality of life. The severity of hemophilia is determined by the level of clotting factor activity in a person’s blood, and there is a negative correlation between risk of bleeding and factor activity levels. Prolonged bleeding episodes can lead to serious complications, such as bleeding into joints, muscles, or internal organs, including the brain. People with hemophilia A or B can develop inhibitors, which prevent their factor VIII or IX replacement treatment from working to form a clot to stop bleeding.

Novo’s FDA submission was based on phase 3 data that met its primary endpoint of reducing spontaneous and traumatic bleeds by 86 percent in a prophylactic setting compared to a control arm.

Photo: Martin Lange, executive vice president of Development, Novo Nordisk