27 February 2023

Two recent advances in haemophilia A have been hailed as “important milestones” in the treatment of the condition.

Firstly, a new formulation of clotting factor VIII has been found effective over a long duration, researchers have reported. The drug, efanesoctocog alfa, is designed to maintain high levels of factor VIII by avoiding the half-life ‘ceiling’ imposed by von Willebrand factor.

A team led by Dr Annette von Drygalski from the University of California San Diego set out to test the efficacy and safety of the drug in previously treated patients with severe haemophilia A.

They recruited patients aged 12 or above with severe haemophilia A and gave one group of 133 patients once-weekly efanesoctocog alfa for a year. The other group of 26 patients received efanesoctocog alfa when needed for six months, then once a week for six months.

In the first group who had regular medication, the estimated mean bleeding rate was 0.69, which was significantly lower compared to that during pre-study Factor VIII prophylaxis of 2.96 (a 77% relative reduction). They also showed improved physical health, pain intensity, and joint health.

In all patients, 97% of bleeding episodes were resolved with one injection of efanesoctocog alfa.

Overall, the drug “had an acceptable side-effect profile”, say the researchers in the New England Journal of Medicine recently, adding: “Once-weekly efanesoctocog alfa provided superior bleeding prevention to prestudy prophylaxis.”


A second recent study has found that the monoclonal antibody emicizumab may be effective for patients with non-severe haemophilia A.

The HAVEN 6 study was led by Dr Claude Négrier from the University Claude Bernard Lyon in France, and results were published in Lancet Haematology. The team found that patients treated with emicizumab had reduced bleeding episodes, and no new concerns regarding safety.

Emicizumab for mild or moderate haemophilia A is now being assessed by NICE for use on the NHS.


Prof Johannes Oldenburg from University Hospital Bonn in Germany, which took part in both studies, said: “Efanesoctocog alfa with its prolonged duration of action, and emicizumab for patients with a moderate course of the disease, represent important milestones in the treatment of haemophilia A.”



von Drygalski A, Chowdary P, Kulkarni R, Susen S, Konkle BA, Oldenburg J, Matino D, Klamroth R, Weyand AC, Jimenez-Yuste V, Nogami K, Poloskey S, Winding B, Willemze A, Knobe K, for the XTEND‑1 Trial Group. (2023) “Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A.” New England Journal of Medicine, doi: 10.1056/NEJMoa2209226

Link: https://www.nejm.org/doi/10.1056/NEJMoa2209226

Négrier C, Mahlangu J, Lehle M, Chowdary P, Catalani O, Bernardi RJ, Jiménez-Yuste V, Beckermann BM, Schmitt C, Ventriglia G, Windyga J, d'Oiron R, Moorehead P, Koparkar S, Teodoro V, Shapiro AD, Oldenburg J, Hermans C. (2023) “Emicizumab in people with moderate or mild haemophilia A (HAVEN 6): a multicentre, open-label, single-arm, phase 3 study.” Lancet Haematology, doi: 10.1016/S2352-3026(22)00377-5

Link: https://www.thelancet.com/journals/lanhae/article/PIIS2352-3026(22)00377-5/fulltext


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