HEMOPHILIA GENE THERAPY MARKET ANALYSIS

Market Challenges And Opportunities

• Serious adverse effects associated with plasma derived products, and limited access to hemophilia treatment

Increasing hemophilia patient base worldwide especially in developed countries is expected to drive growth of the hemophilia gene therapy market:

High prevalence of hemophilia is increasing the demand for sustainable treatment of the disease, which is projected to drive the market growth over the forecast period. According to the National Hemophilia Foundation factsheet 2018, hemophilia affected over 400,000 individuals worldwide. Moreover, according to same source, an estimated 75% of patients suffering from hemophilia have no access to treatment or receive inadequate treatment. According to the Centers for Disease Control and Prevention (CDC) report, Hemophilia A occurs in 1 out of 5,000 live male births. It is around four times more common than Hemophilia B. Few effective treatments available for hemophilia require lifetime infusion of expensive drugs manufactured through recombinant biotechnology or from human plasma.

Furthermore, in September 2019, according to the American Journal of Managed Care, the meta-analysis showed that more than 1,125,000 men around the world have the inherited bleeding disorder; 418,000 of those have a severe version of the mostly undiagnosed disease. Previously, only 400,000 people globally were estimated to have hemophilia.

The study found that, per 100,000 males, there are:

• 1 cases for all severities of hemophilia A
• 0 cases for severe hemophilia A
• 8 cases for all severities of hemophilia B
• cases for severe hemophilia B

Per 100,000 males, the prevalence at birth is:

• 6 cases for all severities of hemophilia A
• 5 cases for severe hemophilia A
• 0 cases for all severities of hemophilia B
• 5 cases for severe hemophilia B

For those born with hemophilia, the chances of living a life of normal duration and quality will be reduced by 64% in upper-middle income countries, 77% in middle income, and up to 93% in low income countries. In 2020, the World Federation of Hemophilia (WFH) identified 165,379 people with haemophilia A and 33,076 people with haemophilia B worldwide.

Moreover, according to Center for Disease Control and Prevention, Hemophilia A affects 1 in 5,000 male births and about 400 babies are born with hemophilia A each year. Figure 2. Global Hemophilia Gene Therapy Market Share (%), by Region, 2022

Strong gene-based product pipeline for hemophilia treatment is expected to drive the hemophilia gene therapy market growth

Gene therapy for hemophilia has strong product pipeline, which is expected to enter the market in the near future. This is expected to drive the market growth, as it would result in availability of new treatment options for management of hemophilia. In Januarys 2019, over 15 investigational gene therapy are under the clinical development stage. In December 2018, Spark Therapeutics announced updated preliminary data for the ongoing phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A.

Current therapies for treating hemophilia are only symptomatic management of the disease by administering clotting factors XIII or IX. Furthermore, there are serious adverse effects associated with plasma derived products, and limited access to treatment. Gene therapy may address these issues to certain level. BioMarin Pharmaceutical’s gene therapy product Valoctocogene roxaparvovec for hemophilia A was in phase III of clinical development in 2019.

Furthermore, in January 2021, the League of Clinical Research, Russia carried out Interventional study for Efmoroctocog Alfa Injection [Eloctate] to treat haemophilia A. The study is currently in Phase 4 and the study is estimated to be completed on November 15, 2022.

Global Hemophilia Gene Therapy Market: Restraint

The major factors that hinder growth of the global hemophilia gene therapy market include serious adverse effects associated with plasma derived products, and limited access to hemophilia treatment. For instance, in September 2019, according to the National Center for Biotechnology Information, a major complication of treatment with coagulation factor concentrates in hemophilia is the development of alloantibodies directed against factor VIII or IX. A common long-term complication of hemophilia is permanent damage to the joints (hemarthropathy) caused by repeated bleeding episodes.