Hemophilia Gene Therapy Market : Get Facts About Business Strategies 2018–2026
Hemophilia Gene
Therapy Market
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.
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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.
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More On Hemophilia
Gene Therapy Market
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