The global 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), was valued at US$ 00.0 million in 2017 and is projected to exhibit
a CAGR of 56.0% over the forecast period (2018 – 2026).
Hemophilia is one of the
prominent genetic disorders that is characterized by the absence of clotting
factors of 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, 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.
Growing patient base for
hemophilia Increasing prevalence of hemophilia worldwide is expected to drive
growth of the hemophilia gene therapy market. In 2016, 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 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 There are numerous pharma and biotech companies that
are engaged in developing hemophilia gene therapy. Most of the companies are
targeting hemophilia A, which has approximately 80% share in overall hemophilia
patients. 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 clinicaltrial.gov, 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”
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Report: https://www.coherentmarketinsights.com/market-insight/hemophilia-gene-therapy-market-2480
There are various steps taken by
the U.S. FDA 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 provides 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.
Key Takeaways of the Hemophilia
Gene Therapy Market:
The global hemophilia gene
therapy market is expected to exhibit a CAGR of 56.0% over the forecast period,
owing to 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, 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. The U.S.
has high annual cost of hemophilia treatment. Current medication regimen to
treat hemophilia costs around US$ 270,000 annually per patient, according to a
2015 Express Scripts report. This cost may be with gene therapy, which is
comparatively sustainable.
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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