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) - Size, Share, Trends, and Forecast to 2026

  • Published On : Feb 2019 |
  • Pages : 110 Pages |
  • Format : CMI Insight PPT Format CMI Insight PDF Format

Hemophilia Gene Therapy Market - Coding For Improved Quality of Life.

Hemophilia is a genetic bleeding disorder in which an individual lacks or has low levels of proteins called clotting factors. There are around 13 types of clotting factors that work with blood platelets, which are necessary for clotting process to initiate. There are three forms of hemophilia - A, B, and C. Hemophilia A is the most common form and is caused due to deficiency in clotting factor VIII. Hemophilia B occurs due to deficiency of clotting factor IX and Hemophilia C is caused due to clotting factor XI deficiency. Hemophilia is incurable with current therapeutic options, which only reduces symptoms such as spontaneous bleeding in muscles and joints as well as increased risk for intracranial hemorrhage. These treatment options lasts only for a day and is costly. As against conventional clotting factor replacement therapy, gene therapy is expected to offer sustainable cure for hemophilia by correcting defective gene sequence (F8 or F9 gene) that codes for clotting factor VIII or IX in the patient’s body.  

In this therapy, recombinant Adeno - associated virus is most commonly utilized to deliver a codon optimized version of the clotting factors genes (VIII or IX) to patients affected by hemophilia. This gene is shortened by deleting a discrete portion of the gene to better fit the coding sequence into the viral vector. There is dose dependent gene expression level observed which means genetic expression for clotting factor increases with increased dose of gene therapy.

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).

Figure 1. Global Hemophilia Gene Therapy Market Value (US$ Mn), by Region, 2017

Hemophilia Gene Therapy  | Coherent Market Insights

Source – Coherent Market Insights Report

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.

Gene therapy may prove to be panacea in this scenario of increasing prevalence rate of hemophilia worldwide.

Figure 2. Global Hemophilia Gene Therapy Market, Product Pipeline, 2017

Company

Product

Indication

Clinical Stage

BioMarin Pharmaceuticals

Valoctocogene

roxaparvovec

Hemophilia A

Phase III

Pfizer

Fidanacogene elaparvovec

Hemophilia B

Phase III

uniQure

AMT-061

Hemophilia B

Phase III

Sangamo Therapeutics

SB-525

Hemophilia A

Phase I/II

Spark Therapeutics

SPK-8011

Hemophilia A

Phase I/II

Shire PLC

SHP 654

Hemophilia A

Phase I

Source – Clinicaltrials.gov, Secondary Analysis by Coherent Market Insights

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.

Hemophilia is a genetic disorder in which patient’s blood do not clot normally due to lack of blood-clotting proteins known as coagulating factors. Conventional treatment options include timely replacement of the specific clotting factor. Major pharma and biotech companies are conducting research and development to develop gene therapy for hemophilia due to various shortcomings with conventional therapy. Increasing number of hemophilic patients that require treatment and strong product pipeline are expected to drive growth of the hemophilia gene therapy market.

Market Dynamics

Major factors that are driving growth of the hemophilia gene therapy market include increasing prevalence of hemophilia, issue of high cost, access to current hemophilia treatment, which includes coagulation factor VIII and IX, and increasing number of companies engaged in research and development of gene therapy products. According to the Centers for Disease Control and Prevention, hemophilia A is a rare, X-linked bleeding disorder that affects around 1 in 5,000 to 10,000 live-born males. Hemophilia B is less common than hemophilia A, with an incidence of around 1 in 25,000 births. According to 2016 global survey by the World Federation of Hemophilia, the total number of individuals with hemophilia worldwide was 184,723, including 16,949 within the U.S.

Key features of the study:

  • This report provides in-depth analysis of the global hemophilia gene therapy market and provides market size (US$ Mn) and compound annual growth rate (CAGR %) for the forecast period (2018 – 2026), considering 2017 as the base year
  • It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrix for this market
  • This study also provides key insights about market drivers, restraints, opportunities, pipeline analysis, market trends,  regional outlook, and competitive strategies adopted by key players
  • It profiles key players in the global hemophilia gene therapy market based on the following parameters – company overview, financial performance, market presence, distribution strategies, key developments and strategies, and future plans
  • Key companies covered as a part of this study include BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC, Sangamo Therapeutics, Inc., and Freeline Therapeutics
  • Insights from this report would allow marketers and the management authorities of the companies to make informed decision regarding their future product launches, product up-gradation, market expansion, and marketing tactics
  • The global hemophilia gene therapy market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
  • Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the hemophilia gene therapy market

Detailed Segmentation:

  • Global Hemophilia Gene Therapy Market, By Type:
    • Hemophilia A
    • Hemophilia B
  • Global Hemophilia Gene Therapy Market, By Region:
    • North America
      • By Type
        • Hemophilia A
        • Hemophilia B
      • By Country:
        • U.S.
        • Canada
    • Latin America
      • By Type
        • Hemophilia A
        • Hemophilia B
      • By Country:
        • Brazil
        • Mexico
        • Argentina
        • Rest of Latin America
    • Europe
      • By Type
        • Hemophilia A
        • Hemophilia B
      • By Country:
        • Germany
        • U.K.
        • France
        • Italy
        • Spain
        • Russia
        • Rest of Europe
    • Asia Pacific
      • By Type
        • Hemophilia A
        • Hemophilia B
      • By Country:
        • China
        • India
        • Japan
        • Australia
        • South Korea
        • ASEAN
        • Rest of Asia Pacific
    • Middle East
      • By Type
        • Hemophilia A
        • Hemophilia B
      • By Country:
        • GCC
        • Israel
        • Rest of Middle East
    • Africa
      • By Type
        • Hemophilia A
        • Hemophilia B
      • By Country:
        • South Africa
        • Central Africa
        • North Africa
  • Company Profiles
    • BioMarin Pharmaceuticals, Inc. *
      • Company Overview
      • Product Portfolio
      • Key Highlights
      • Financial Overview
      • Market Strategies
    • Spark Therapeutics
    • Pfizer, Inc.
    • UniQure NV
    • Ultragenyx Pharmaceutical
    • Shire PLC
    • Sangamo Therapeutics
    • Freeline Therapeutics

“*” marked represents similar segmentation in other categories in the respective section.

Table of Contents

  1. Research Objectives and Assumptions
    • Research Objectives
    • Assumptions
    • Abbreviations
  2. Market Purview
    • Report Description
      • Market Definition and Scope
    • Executive Summary
      • Market Snippet, By Hemophilia Type
      • Market Snippet, By Region
    • Coherent Opportunity Map (COM)
  3. Market Dynamics, Regulations, and Trends Analysis
    • Market Dynamics
      • Drivers
      • Restraints
      • Market Opportunities
    • Impact Analysis
    • Pipeline Analysis
    • Primary Research Respondents View About Market
    • Mergers, Acquisitions, and Collaborations
    • Regulatory Scenario
    • PEST Analysis
    • Epidemiological Data
  4. Global Hemophilia Gene Therapy Market, By Product Type, 2018 – 2026, (US$ Mn)
    • Introduction
      • Market Share Analysis, 2017 and 2025 (%)
      • Y-o-Y Growth Analysis, 2017 – 2026
      • Segment Trends
    • Hemophilia A
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2016 – 2026, (US$ Mn)
    • Hemophilia B
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2016 – 2026, (US$ Mn)
  5. Global Hemophilia Gene Therapy Market, By Region, 2018 – 2026, (US$ Mn)
    • Introduction
      • Market Share Analysis, By Region, 2017 and 2026 (%)
      • Y-o-Y Growth Analysis, For Regions, 2017 – 2026
    • North America
      • Market Size and Forecast, By Hemophilia Type, 2016 – 2026, (US$ Mn)
      • Market Size and Forecast, By Country, 2016 – 2026, (US$ Mn)
        • U.S.
        • Canada
    • Latin America
      • Market Size and Forecast, By Hemophilia Type, 2016 – 2026, (US$ Mn)
      • Market Size and Forecast, By Country, 2016 – 2026, (US$ Mn)
        • Brazil
        • Mexico
        • Argentina
        • Rest of Latin America
    • Europe
      • Market Size and Forecast, By Hemophilia Type, 2016 – 2026, (US$ Mn)
      • Market Size and Forecast, By Country, 2016 – 2026, (US$ Mn)
        • Germany
        • U.K.
        • France        
        • Italy
        • Spain
        • Russia
        • Rest of Europe
    • Asia Pacific
      • Market Size and Forecast, By Hemophilia Type, 2016 – 2026, (US$ Mn)
      • Market Size and Forecast, By Country, 2016 – 2026, (US$ Mn)
        • China
        • India
        • Japan
        • Australia
        • South Korea
        • ASEAN
        • Rest of Asia Pacific
    • Middle East
      • Market Size and Forecast, By Hemophilia Type, 2016 – 2026, (US$ Mn)
      • Market Size and Forecast, By Country, 2016 – 2026, (US$ Mn)
        • GCC
        • Israel
        • Rest of Middle East
    • Africa
      • Market Size and Forecast, By Hemophilia Type, 2016 – 2026, (US$ Mn)
      • Market Size and Forecast, By Country, 2016 – 2026, (US$ Mn)
        • South Africa
        • Central Africa
        • North Africa
  6. Competitive Landscape
    • Market Share Analysis
    • Company Profiles
      • BioMarin Pharmaceuticals, Inc.*
        • Company Overview
        • Product Portfolio
        • Financial Performance
        • Recent Developments/Updates
        • Future Plans
      • Spark Therapeutics
        • Company Overview
        • Product Portfolio
        • Financial Performance
        • Recent Developments/Updates
        • Future Plans
      • Pfizer, Inc.
        • Company Overview
        • Product Portfolio
        • Financial Performance
        • Recent Developments/Updates
        • Future Plans
      • UniQure NV
        • Company Overview
        • Product Portfolio
        • Financial Performance
        • Recent Developments/Updates
        • Future Plans
      • Ultragenyx Pharmaceutical
        • Company Overview
        • Product Portfolio
        • Financial Performance
        • Recent Developments/Updates
        • Future Plans
      • Shire PLC
        • Company Overview
        • Product Portfolio
        • Financial Performance
        • Recent Developments/Updates
        • Future Plans
      • Sangamo Therapeutics, Inc
        • Company Overview
        • Product Portfolio
        • Financial Performance
        • Recent Developments/Updates
        • Future Plans
      • Freeline Therapeutics
        • Company Overview
        • Product Portfolio
        • Financial Performance
        • Recent Developments/Updates
        • Future Plans
    • Analyst Views
  7. Section
    • References
    • Research Methodology
    • About us and Sales Contact

*Browse 16 market data tables and 21 figures on "Hemophilia Gene Therapy Market - Global forecast to 2026”.

Research Methodology

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