Pharmaceutical company Novo Nordisk has announced positive results for its prophylactic treatment targeting people living with haemophilia A or B with inhibitors.
The company’s concizumab treatment is an anti-tissue factor pathway inhibitor (TFPI) antibody in development to prevent prolonged and spontaneous bleeding in all types of haemophilia.
Results from Novo Nordisk’s Phase III explorer71 study showed an 86% reduction in treated spontaneous and traumatic bleeds when on concizumab prophylaxis, with an estimated mean average bleeding rate (ABR) of 1.7 compared to 11.8 for patients with no prophylaxis. During the study, 21 people on concizumab experienced no treated bleeds, compared to two (10.5%) on no prophylaxis.
Lead investigator Dr Victor Jiménez-Yuste, MD, Haematology Department, La Paz University Hospital, Madrid, Spain, said: “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. 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 in the EU and the UK.
1: Jiménez-Yuste V, Angchaisuksiri P, Castaman G, et al. Concizumab prophylaxis in patients with haemophilia A or B with inhibitors: Efficacy and safety results from the 32-week primary analysis of the phase 3 explorer7 trial – ISTH 2022, late breaking abstract available 9-13 July