Hemophilia A is an X-linked congenital disorder, characterized by factor VIII (FVIII) deficiency [1, 2]. Hemophilia A manifests as spontaneous bleeding into the muscles and joints, which, if inadequately managed, becomes painful and destructive to joint architecture [1, 2].

Global survey data from the World Federation of Hemophilia (WFH) indicate that India has the largest population of individuals with hemophilia A worldwide, with 19,690 cases (incidence: 1.4 per 100,000 people) reported in 2020, followed by the United States, with 13,915 cases (4.2 per 100,000 people) [3]. By comparison, the incidence of hemophilia A varies across the Asian region, with 0.8 cases per 100,000 in Indonesia (n = 2214) and 1.1 cases per 100,000 in the Philippines (n = 1155) compared with 2.2 per 100,000 in Thailand (n = 1557) and 3.0 per 100,000 in Malaysia (n = 950) [3].

In India, a large proportion of individuals with hemophilia either remain undiagnosed or present for treatment at a late stage, with many cases unregistered. Consequently, the actual prevalence of hemophilia A is probably underestimated, and when individuals are diagnosed, they typically have more severe disease [4]. Based on these data, India carries a disproportionate amount of the global hemophilia A health care burden, signifying an unmet need for appropriate and effective treatment for this patient population. The current standard of care encompasses prophylactic or on-demand treatment with recombinant or plasma-derived clotting factor concentrate, or prophylaxis with a monoclonal antibody, such as emicizumab [1].

For those with moderate or severe hemophilia A, intravenous replacement with exogenous FVIII is typically used to prevent or treat hemorrhage [1]. Treatment for hemophilia accounts for the majority of the overall cost of management, with clotting factor concentrates accounting for more than 86% of direct costs in individuals without inhibitors [5]. High treatment costs are recognized as a barrier for access to treatment. However, the beneficial quality-of-life outcomes associated with prophylaxis are likely to outweigh the cost of treatment [5,6,7].

Despite the prevalence of hemophilia A, the use of FVIII (both standard and extended half-life products) is much lower in India than in many other parts of Asia and the rest of the world [3]. Recent 2020 WFH estimates for mean per capita FVIII use in 2019 were 0.289 IU/total population in India compared with estimates ranging from 0.622 IU/total population for Thailand to 2.219 in Singapore, 7.105 in the United States, and 9.026 in the United Kingdom [3]. Moroctocog alfa (AF-CC) is a third-generation recombinant FVIII produced using advanced manufacturing and purification, and is free of exogenous human- and animal-derived protein [8,9,10,11]. It is approved in India for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in participants with hemophilia A (congenital FVIII deficiency or classic hemophilia) [12, 13]. This post-approval study evaluated the safety and efficacy of moroctocog alfa in participants with moderate or severe congenital hemophilia A in India.