Novo Nordisk’s Alhemo (concizumab) has been recommended by the European Medicines Agency’s human medicines committee to prevent bleeding in haemophilia patients aged 12 years or older.
If approved, Alhemo would be the first once-daily subcutaneous prophylactic treatment for patients with haemophilia A or B and inhibitors.
Affecting approximately 1,125,000 people globally, haemophilia is a rare inherited bleeding disorder that impairs the body’s ability to make blood clots, a process needed to stop bleeding.
The condition is usually treated with regular intravenous infusions of clotting factor replacement products. However, some patients can develop inhibitors as an immune response to the clotting factors in these therapies, limiting treatment options.
It is estimated that up to 30% of patients with severe haemophilia A and up to 10% of those with severe haemophilia B develop inhibitors.
Novo’s Alhemo is an anti-tissue factor pathway inhibitor monoclonal antibody that is designed to help the blood to clot and prevent bleeding, even in the presence of inhibitors.
The Committee for Medicinal Products for Human Use’s decision on the therapy was supported by positive results from the late-stage explorer7 study, which demonstrated an 86% reduction in treated spontaneous and traumatic bleeds in patients receiving Alhemo prophylaxis compared to no prophylaxis.
Novo said it expects the European Commission to make a decision on Alhemo within approximately two months.
Stephanie Seremetis, the company’s chief medical officer for haemophilia, described the positive opinion as a “major milestone for patients”.
She said: “[Alhemo] could alleviate the physical, emotional and overall treatment burden for people living with haemophilia, as it is delivered in a pre-filled, multi-use, portable pen that can be stored at room temperature for up to four weeks.
“This could enable patients to have greater confidence in the pursuit of daily activities, which is especially important for people living with haemophilia B with inhibitors, who currently have very limited treatment options.”
The recommendation comes just one month after Novo entered into a multi-year partnership with NanoVation Therapeutics to advance genetic medicines for cardiometabolic and rare diseases.
The deal, which is worth worth $600m, combines Novo’s capabilities in the two areas with NanoVation’s proprietary long-circulating lipid nanoparticle technology for RNA delivery to cells outside of the liver.