Market Trends of Hemophilia Industry
Hemophilia A is Expected to Hold Significant Market Share in the Disease Type Segment
Hemophilia A is the most common severe bleeding disorder. Replacement therapy, providing the missing coagulation factor, was the mainstay of treatment both prophylactically and to treat bleeding. Hemophilia A is expected to hold a significant market share over the forecast period owing to factors such as an increase in the prevalence of hemophilia A cases, a rise in awareness programs, and a surge in different therapies for hemophilia A. For instance, according to the World Federation of Hemophilia Report on the Annual Global Survey 2021, 233,577 people were living with hemophilia in 2021 globally, out of which 185,318 cases were hemophilia A.
Furthermore, product approval, clearance, and launches will likely increase market opportunities over the forecast period. For instance, in August 2022, the European Commission (EC) granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under ROCTAVIAN. BioMarin includes multiple clinical studies underway in its comprehensive gene therapy program for treating severe hemophilia A. Similarly, in June 2022, the FDA granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for treating people with hemophilia A. It is a rare, life-threatening bleeding disorder based on pivotal XTEND-1 Phase 3 study data. Sanofi and Sobi collaborated on the development and commercialization of efanesoctocog alfa.
Moreover, research and development to advance the treatment for hemophilia A are widely carried out, which is expected to fuel the market growth over the forecast period. For instance, as per the article published in September 2022 in PubMed, various key players are working on an in vivo genome-editing program based on CRISPR/Cas9. This technique uses non-homologous end-joining to prevent the loss of the AAV vector due to hepatocyte growth. It is to enable the permanent chromosomal integration of a modified human B-domain-deleted FVIII at the albumin locus in liver cells. Young individuals with hemophilia A who are currently ineligible for traditional gene therapy may benefit greatly from such a method.
Therefore, due to the increase in hemophilia A cases and the rise in different pipeline studies and product approvals for hemophilia A, the studied segment is expected to include significant market growth over the forecast period.
North America Expected to Hold Significant Share in the Market and Expected to do Same Over the Forecast Period
North America is expected to hold a significant share of the market. It is expected to do the same over the forecast period due to factors such as the rise in hemophilia cases, increasing awareness about technologically advanced products, and favorable regional government initiatives. For instance, according to the CDC update in August 2022, about 33,000 people in the US contain hemophilia. Hemophilia typically affects men and people assigned male at birth (AMAB). As per the same source, the estimated prevalence of hemophilia in the United States is 12 cases per 100,000 US males for hemophilia A and 3.7 cases per 100,000 US males for hemophilia B.
Furthermore, according to the study published in Frontiers in Immunology in April 2021, in the United States, patients with hereditary disorders, such as hemophilia B, benefited from Adeno-associated virus (AAV)-mediated gene transfer by attaining long-term expression of the therapeutic transgene. Such studies further bolster gene therapies for hemophilia, thereby driving market growth. Moreover, rising initiatives from key market players are also expected to drive market growth in this region. For instance, in February 2022, BioMarin Pharmaceutical Inc. presented positive results from a two-year Phase 3 GENEr8-1 study analysis and an overall safety update of valoctocogene roxaparvovec. It is an investigational gene therapy for treating adults with severe hemophilia A at the 15th Annual Virtual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD).
Additionally, approvals and clearances for hemophilia-related products and therapies are expected to bolster market growth over the forecast period. For instance, in October 2022, Novo Nordisk received Health Canada approval for a new indication for REBINYN (Coagulation Factor IX (Recombinant), pegylated) for the treatment of routine prophylaxis in hemophilia B patients that are <18 years of age. REBINYN is now indicated in all ages with hemophilia B (congenital factor IX deficiency or Christmas disease) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes and control and prevent bleeding in a perioperative setting.
Thus, North America is expected to include a significant market share over the forecast period due to the increase in hemophilia cases and the rise in different therapies for hemophilia coupled with research and development.
