The hematological disorders market is undergoing an impressive surge in sales, cementing its position as the fastest-growing therapy area for the second consecutive year. Notably, this growth is not evenly distributed, with 45% of the sales growth being driven by just three drugs: Alexion Pharmaceuticals’s Ultomiris (ravulizumab-cwvz), Genentech’s Hemlibra (emicizumab-kxwh), and Akebia Therapeutics’s Vafseo (vadadustat), according to GlobalData, a leading data and analytics company.

GlobalData’s Looking Ahead to 2023 report reveals that hematology sales are highly dependent on event-driven outcomes in these key drugs, including regulatory approvals and the release of positive clinical trial data.

Kevin Marcaida, Pharma Analyst at GlobalData, comments: “The hematological disorders market will experience significant growth of 16% from 2022 to 2023, followed by oncology and metabolic disorders with 12% and 9% growth, respectively.”

Ultomiris, a humanized monoclonal antibody complement inhibitor, is the most prominent drug driving hematology sales, accounting for 21% of the market’s growth in 2023. This can be attributed to the drug successfully gaining two approvals in the US, specifically for the treatment of atypical hemolytic uremic syndrome and paroxysmal nocturnal hemoglobinuria in June 2022.

Hemlibra is a bispecific monoclonal antibody designed to provide the cofactor function of factor VIII in people with hemophilia A. It is currently the best-selling hematology drug, with sales of $4.2 billion in 2022. It accounts for 16% of hematology sales growth, which is driven by a supplemental approval for hemophilia A in the EU in January 2023.

This approval was subsequently expanded the following month to include hemophilia A without factor VIII inhibitors, which could result in an increase in sales forecasts. Hemlibra also gained regulatory approval in Japan for acquired hemophilia A in June 2022.

Vafseo is an oral hypoxia-inducible factor-prolyl hydroxylase (HIF-PH) inhibitor for the treatment of anemia in chronic kidney disease. In 2023, Vafseo accounts for 7% in hematology sales growth. This makes Vafseo the fastest-growing drug in the hematological disorders market by far. This growth is driven by its regulatory approval in the EU for anemia in chronic kidney disease in April 2023.

The approval was based on data obtained from the global Phase III INNO2VATE clinical program. The data showed that Vafseo achieved the primary efficacy endpoint defined by its superiority, compared with darbepoetin alfa, in the time elapsed before a first major adverse cardiovascular event.

Marcaida concludes: “The concentration of sales growth in a few key drugs means that the near-term outlook for the hematological disorders market is reliant on event-driven outcomes such as regulatory approvals and clinical trial results. This dynamic may change over time if new drugs are approved to diversify sales growth. However, in the short-term, the market will likely be driven by sales growth in Ultomiris, Hemlibra, and Vafseo. With few drugs driving growth, there is a need for pharmaceutical companies to focus on developing innovative therapies that can meet other medical needs within the field.”