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.
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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.
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.
Detailed
Segmentation:
Global
Hemophilia Gene Therapy Market, By Type:
-
Hemophilia A
-
Hemophilia B
Global
Hemophilia Gene Therapy Market, By Region:
-
North America
-
Latin America
-
Europe
-
Asia Pacific
-
Middle East
-
Africa
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Insights Of Global Hemophilia Gene Therapy Market Research Report At
https://www.coherentmarketinsights.com/market-insight/hemophilia-gene-therapy-market-2480
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