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Hemophilia Gene Therapy Market Foreseen to Exceed the US$ 780.0 Million by 2026 | CAGR: 56.0%: Coherent Market Insights

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The global hemophilia gene therapy marketby Hemophilia Type (Hemophilia A and Hemophilia B) and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) is projected to exhibit a CAGR of 56.0% during the forecast period (2018 – 2026) as highlighted in a new report published by Coherent Market Insights.

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Hemophilia is one of the prominent genetic disorders that is characterized by the absence of clotting factors of a specific type resulting in lack of coagulation after blood loss. This may result in serious health complications. Bleeding may be due to injury to external tissue or spontaneous bleeding in muscles and joints. Existing therapy offers symptomatic approach where clotting factors that are absent are provided externally on regular basis. However, there are many limitations of this approach such as toxicity, short term efficacy, high cost, and accessibility. Gene therapy offers solutions to all these limitations. Most important is the ability of the hemophilia gene therapy to address the root cause of hemophilia which is caused due to genetic mutations of the gene that codes for the clotting factors VIII (Hemophilia A) and clotting factors IX (Hemophilia B). In gene therapy, a specific gene is inserted into viral vectors (rAAV is most commonly used as vectors) and this vector-based gene is injected intravenously into hemophilic patients, which then codes for formation of coagulation factors.

The growing patient base for hemophilia

The increasing prevalence of hemophilia worldwide is expected to drive growth of the hemophilia gene therapy market. In 2016, the World Federation of Hemophilia stated that 1 in 5,000 people are affected by hemophilia A, and 1 in 50,000 are affected by hemophilia B. According to the National Hemophilia Foundation factsheet 2018, over 400,000 individuals were affected by hemophilia worldwide. Furthermore, according to the 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), the median age for diagnosis is one month for individuals with severe symptoms, 8 months for individuals with moderate hemophilia, and 36 months for people with mild hemophilia.

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Strong product pipeline for hemophilia gene therapy

Numerous pharma and biotech companies are engaged in developing hemophilia gene therapy. Most of the companies are targeting hemophilia A, which accounts for 80% of the overall hemophilia incidence. BioMarin Pharmaceuticals’ investigational gene therapy, Valoctocogene roxaparvovec for treating hemophilia A, is currently in phase III, and Sangamo therapeutics’ investigational gene therapy SB – 525 is in phase I/II, which gave positive preliminary data in August 2018. In December 2018, Spark Therapeutics announced updated preliminary data for the ongoing phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A. Shire PLC’s gene therapy SHP654 for hemophilia A is currently in phase I of clinical development. It is expected that the first gene therapy would enter the market by 2022. Overall, as per database of, there are around 17 investigational gene therapies under development. Although hemophilia gene therapy may be accessible to only limited number of patients initially due to uniqueness of therapy, it may be widely used over the period, majorly in emerging economies in Asia Pacific and the Middle East, where coagulation factors replacement therapy has limited access.

Browse 16 Market Data Tables and 21 Figures spread through 110 Pages and in-depth TOC on “Hemophilia Gene Therapy Market – by Hemophilia Type (Hemophilia A and Hemophilia B) and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa)– Global forecast to 2026”

To know the latest trends and insights prevalent in the hemophilia gene therapy market press release, click the link below:

The U.S. FDA is taking several steps to speed up the clinical development of hemophilia gene therapy. The U.S. FDA has allotted breakthrough therapy designation to several investigational gene therapy. For instance, in 2017, the U.S. Food and Drug Administration granted breakthrough therapy designation to valoctocogene roxaparvovec of BioMarin Pharmaceuticals. Inc. Earlier in 2016, company received orphan drug designation for valoctocogene roxaparvovec. The U.S FDA also announced new draft guidelines in 2018 that provide information about the required design and other parameters for an accelerated approval approach, which would help the developers of human gene therapy (GT) products for the treatment of hemophilia A & B.

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Key Takeaways of the Hemophilia Gene Therapy Market:

  • The global hemophilia gene therapy market is expected to exhibit a CAGR of 56.0% during the forecast period owing to the increasing prevalence of hemophilia worldwide, high cost and limitations with the current clotting factor replacement therapy, and high number of investigational gene therapy under clinical development stage
  • Among hemophilia type, the hemophilia A segment is expected to hold dominant position in the market due to high prevalence rate amongst population worldwide
  • North America is expected to hold dominant position in the market, followed by Europe and Asia Pacific as the U.S. has a high annual cost of hemophilia treatment. Current medication regimes to treat hemophilia cost around US$ 270,000 annually per patient, according to a 2015 Express Scripts report.
  • Key players operating in the global hemophilia gene therapy market are BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC, Sangamo Therapeutics, Inc., and Freeline Therapeutics.

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Coherent Market Insights is a global market intelligence and consulting organization focused on assisting our plethora of clients achieve transformational growth by helping them make critical business decisions. We are headquartered in India, having office at global financial capital in the U.S. and sales consultants in United Kingdom and Japan. Our client base includes players from across various business verticals in over 150 countries worldwide. We pride ourselves in catering to clients across the length and width of the horizon, from Fortune 500 enlisted companies, to not-for-profit organization, and startups looking to establish a foothold in the market. We excel in offering unmatched actionable market intelligence across various industry verticals, including chemicals and materials, healthcare, and food & beverages, consumer goods, packaging, semiconductors, software and services, Telecom, and Automotive. We offer syndicated market intelligence reports, customized research solutions, and consulting services.

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Last Updated: 08-Oct-2020