FDA rejects consizumab for prophylactic treatment of hemophilia A, B

Pharmaceutical company Novo Nordisk said in its first-quarter earnings call that the US Food and Drug Administration (FDA) had rejected an application for its investigational antibody, consizumab.¹

According to Martin Lange, Novo’s executive vice president and head of development, the FDA is requesting additional information about the drug’s manufacturing process and the company’s systems “to monitor and administer patients to ensure proper treatment.” I asked.

Concizumab is proposed for the prophylactic treatment of hemophilia A and B. This treatment is a monoclonal antibody that targets the tissue factor pathway inhibitor protein involved in clotting.²

Condizumab, an anti-tissue factor pathway inhibitor (TFPI) antibody, was designed as a once-daily prophylactic treatment administered subcutaneously for all hemophilia types.

In July 2022, Novo published positive Phase 3 data from the Explorer7 trial showing strong results for consizumab in reducing spontaneous and traumatic bleeding.

The data, presented at last year’s International Society on Thrombosis and Haemostasis Annual Meeting (ISTH 2022), show an 86% reduction in treated spontaneous and traumatic bleeding when patients with inhibitors receive consizumab prophylaxis. I showed that.

In 2020, Novo suspended three trials of consizumab after three patients developed non-fatal thrombotic events, then resumed a Phase 3 trial of the drug after working with regulators .

The hemophilia market is currently dominated by Roche’s emicizumab (hemlibra). It is a bispecific antibody against factors IXa and X designed to activate the natural clotting cascade and restore the blood clotting process in hemophilia A patients.³

1. ^{bio space. Novo Nordisk’s hemophilia candidate hits her FDA roadblock. The company cuts his Wegovy supply. bio space. May 3, 2023.}
2. ^{Glossy, Juliana. Concizumab reduces treatment bleeding in hemophilia A or B by inhibitor. HCP Live. July 15, 2022.}
3. ^{Kunzmann, Kevin. Hemlibra shows efficacy in more hemophilia A after FDA approval. HCP Live. November 20, 2017. https://www.hcplive.com/view/hemlibra-shows-more-hemophilia-a-efficacy-after-fda-approval}