The European Medicines Agency (EMA) is recommending that human coagulation factor X (Coagadex) should be authorised to treat the rare bleeding disorder factor X deficiency, which can be fatal.
Patients with this inherited condition can have prolonged bleeding after surgery or injury because they fail to produce the necessary factor X, which allows the blood to coagulate. The current replacement therapy options have associated risks around dosing and clotting that can trigger complications.
Human coagulation factor X – a protein derived from human plasma – is indicated for the treatment and preventive management of bleeding episodes in this group of patients. It has orphan drug status in recognition of its potential to help patients with rare diseases.
The EMA’s Committee for Medicinal Products for Human Use assessed the product under its accelerated assessment procedure because of the unmet clinical need. It based its recommendation on the results of two non-randomised, open-label, multi-centre studies. A phase III study involving 207 spontaneous bleeds in 16 patients with moderate or severe factor X deficiency found an “excellent” or “good” response to treatment with human coagulation factor X in 98.8% of the bleeds treated. Five patients underwent seven surgical procedures and the therapy was effective for the prevention of bleeding during planned surgery.
The recommendation now goes to the European Commission for approval. If marketing authority is given it will be up to individual European Union states to decide the treatment price and level of reimbursement within their own national health systems.