Europe Drug Regulator's Advising Panel Recommends Approval Of Novo Nordisk's Australia-Approved Hemophilia Drug
On Friday, Novo Nordisk A/S said that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending approval of Alhemo (concizumab) as the first once-daily subcutaneous prophylactic treatment for hemophilia patients aged 12 years or older, with A or B with inhibitors.
Haemophilia is a rare bleeding disorder that impairs the body’s ability to make blood clots, a process needed to stop bleeding.
Alhemo is an anti-tissue factor pathway inhibitor (TFPI) monoclonal antibody that, if approved by the European Commission, will offer the first once-daily subcutaneous prophylactic treatment to people with hemophilia A or B with inhibitors. Alhemo is designed to block a protein called TFPI in the body that stops blood from clotting. By blocking TFPI, Alhemo ensures the production of thrombin, which helps to clot the blood and prevent bleeding, even when the other clotting factors are missing or deficient.
The CHMP positive opinion is based on phase 3 explorer7 study data. The results showed an 86% reduction in treated spontaneous and traumatic bleeds when on concizumab prophylaxis, with an estimated mean annualized bleeding rate (ABR) of 1.7 compared to 11.8 with no prophylaxis.
If approved, Alhemo will be provided in a portable, pre-mixed, and prefilled pen, enabling quick and easy subcutaneous administration. Novo Nordisk expects a final approval by the European Commission within approximately two months.
Alhemo is currently approved in Australia and Switzerland for adolescents and adults with hemophilia A or B with inhibitors. In Japan, Alhemo is currently approved for adolescents and adults with hemophilia A or B with and without inhibitors and is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes.
