Hemophilia Gene Therapy Market Size Projected to be Resilient by 2026
Hemophilia Gene Therapy Market – Size, Share, Outlook, and Opportunity Analysis 2018 - 2026
The global hemophilia gene therapy market was valued at US$ 0.00 million in 2017 and is expected to witness a CAGR of 56.0% over the forecast period (2018 – 2026).
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 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.
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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. By 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 is in phase III of clinical development in 2019.
Regulatory bodies such as the U.S. Food and Drug Administration (FDA) are supporting the product development for gene therapy by allotting breakthrough designation status to investigational gene therapy. For instance, in 2017, the U.S. FDA granted breakthrough therapy designation to valoctocogene roxaparvovec, which is an Adeno -associated virus 5 factor VIII vector designed to restore factor VIII plasma concentrations in patients with hemophilia A.
Key players operating in the global hemophilia gene therapy market include BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC Sangamo Therapeutics, Inc., and Freeline Therapeutics.
Global Hemophilia Gene Therapy Market, By Type:
• Hemophilia A
• Hemophilia B
Global Hemophilia Gene Therapy Market, By Region:
• North America
• Latin America
• Asia Pacific
• Middle East
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