Data shows a significant reduction in treated spontaneous and traumatic bleeds among haemophilia patients
Novo Nordisk has announced the phase 3 results of the explorer7 study, assessing the efficacy and safety of prophylactic treatment with concizumab in people living with haemophilia A or B.
The results from the primary analysis were presented at the International Society of Thrombosis and Haemostasis Annual Congress in London.
Concizumab is an anti-tissue factor pathway inhibitor antibody in development for once-daily prophylactic treatment by administration under-the-skin for all types of haemophilia.
The results showed an 86% reduction in treated spontaneous and traumatic bleeds when on concizumab prophylaxis. The safety and tolerability profile of the treatment was also within the expected range, with no thromboembolic events reported after the treatment was restarted.
“One of the most critical complications in the treatment of haemophilia is the development of inhibitors, as they render standard replacement therapy ineffective and severely limit treatment options for haemophilia B,” reflected explorer7 lead investigator Dr Victor Jiménez-Yuste.
He added: “Based on the results of the explorer7 study, there is a potential for concizumab to become a new treatment option for people living with haemophilia A or B with inhibitors.”
“The treatment of haemophilia is complex and no one treatment fits all,” added Martin Lange, executive vice president and head of development at Novo Nordisk. “Concizumab offers the potential for everyday protection for people living with haemophilia and provides an important potential addition to our haemophilia offering, especially in the haemophilia B with inhibitor population who currently have limited treatment options.”
Novo Nordisk expects to submit concizumab for regulatory approval for the prophylactic treatment of haemophilia A or B with inhibitors in the second half of 2022 in the US and Japan and – in 2023 – the EU and the UK.
The use of concizumab is currently investigational and not approved by regulatory authorities.