On 21 June, former students began giving accounts at a public inquiry regarding events at Lord Mayor Treloar College in Hampshire, UK, where at least 72 children with haemophilia died during the 1970s and 1980s after being treated with blood contaminated with human immunodeficiency virus (HIV) and hepatitis. Following this incident, advancements in the manufacturing of recombinant proteins have provided haemophiliacs with therapeutic options that avoid having to receive blood from donors. With developers focusing on improving recombinant products and the advent of gene therapies for haemophilia, GlobalData anticipates a decreased uptake of plasma-derived treatments in haemophilia patients over the next decade.

Haemophilia is a rare, X-linked hereditary disorder in which deficiencies in clotting factors—factor VIII (haemophilia A) or IX (haemophilia B)—result in excess bleeding in joints, muscles and soft tissues after internal or external trauma. Treatments for the disorder were previously restricted to receiving blood plasma from donors who have working levels of these clotting factors. The events at Treloar’s College, however, have permanently altered the haemophilia treatment landscape, sparking the need for mandatory screening for and inactivating of viruses in donated blood. This has minimised contamination with plasma-derived treatments.

Advancements in DNA technology have led to the production of recombinant factors VIII and IX, with the first recombinant product gaining marketing authorisation in the UK in the mid-1990s. There are currently 31 agents marketed in the UK for the treatment of haemophilia A and B. Of these, 48% are either recombinant or synthetic molecules. According to GlobalData’s patient-based forecasts, the total sales of plasma-derived factor concentrates in Europe will fall to $264m and the total sales of recombinant factor concentrates in Europe to $1.4bn by 2028 (Table 1). Two new classes of drug are set to disrupt the market.

Roche’s Hemlibra (emicizumab) is an alternative coagulation promoter that does not have the same structure as factor VIII, making it a valid treatment option for patients with anti-VIIIa neutralising inhibitors. According to GlobalData’s patient-based forecasts, this class of drugs is predicted to have a compound annual growth rate (CAGR) of 37.5% for total sales in Europe between 2018 and 2028 (Table 1). In addition, Hemlibra is more long-acting, with a half-life of four weeks compared to Baxalta’s Feiba’s (anti-inhibitor coagulant complex) half-life of four to seven hours, thereby reducing the number of injections required. Key opinion leaders believe therapies that reduce the frequency of injections will diminish the treatment burden on patients, and that these drugs are set to dominate the market.

Gene therapies, such as UniQure’s etranacogene dezaparvovec, are long-acting drugs that deliver clotting factor genes to cells to replace mutant genes and restore clotting factor plasma concentrations to an adequate level for normal clotting. Earlier this week, a UniQure press release announced that patients in the HOPE-B pivotal trial had demonstrated sustained increases in factor IX activity at 52 weeks post-infusion with a mean activity of 41.5% of normal, thus meeting its co-primary endpoint. UniQure and its partner, CSL Behring, are expected to submit a biologics licensing application (BLA) to the US Food and Drug Administration (FDA) in the first quarter of next year, and the company believes that its candidate could be the first marketed gene therapy to treat haemophilia B.

Cell & Gene Therapy Coverage on Pharmaceutical Technology supported by Cytiva.

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