Lysosomal storage diseases (LSDs)
are a group of over 50 disorders, each involving a different lysosomal enzyme.
According to the National Organization for Rare Disorders, LSDs are believed to
have a frequency of one in every 50,000 live births. Most LSDs are caused due
to autosomal recessive inheritance leading to genetic mutations resulting in deficiency
of lysosomal enzymes.
The global Lysosomal
Storage Diseases Therapeutics Market is estimated to account for US$
7,217.9 Mn in terms of value in 2019 and is expected to reach US$ 15,734.5 Mn
by the end of 2027.
Global Lysosomal Storage Diseases
Therapeutics Market: Drivers
Favorable regulations for orphan
products development are expected to propel boost growth of the global
lysosomal storage diseases therapeutics market over the forecast period. Over
the last few years, introduction of specific orphan drug regulations has
enabled and accelerated research and development of therapies for treatment of
several rare diseases. For instance, the USFDA Office of Orphan Products
Development (OOPD) focuses on advancing the assessment and development of
products (drugs, biologics, devices, or medical foods) that demonstrate
potential for diagnosis and/or treatment of rare diseases.
Global Lysosomal Storage Diseases
Therapeutics Market: Opportunities
Use of nanotechnology is expected
to offer lucrative growth opportunities for players in the global lysosomal
storage diseases therapeutics market. For instance, in October 2019,
researchers from University of Santiago de Compostela, Spain, reported
development of a cytocompatible formulation based on nanostructured solid lipid
carrier systems to deliver the enzyme efficiently to target tissues and
successively preserve its activity inside the target cells, chondrocytes, in
order to improve treatment of LSDs.
* The sample copy includes: Report Summary, Table of
Contents, Segmentation, Competitive Landscape, Report Structure, Methodology.
Request a sample copy
of this report: https://www.coherentmarketinsights.com/insight/request-sample/3651
Global Lysosomal Storage Diseases
Therapeutics Market: Restraints
Lack of treatment options are
expected to limit growth of the market. There exists a significant challenge in
transforming scientific discoveries into new treatment options for orphan
diseases, due to rarity of these diseases and the challenges associated with
conducting clinical trials. Thus, development of new treatment options remains
a challenge and also, absence of effective treatments lessens readiness to
diagnose and eagerness to study further about the disease.
Key Takeaways:
Enzyme replacement therapy
segment in the global lysosomal storage diseases therapeutics market was valued
at US$ 6,856.2 Mn in 2019 and is expected to reach US$ 14,938.7 Mn by 2027 at a
CAGR of 10.0% during the forecast period. eing an orphan disease condition,
regulatory agencies offer the benefit of rapid approval and ease in approval
process. This in turn, is expected to expand the overall enzyme replacement
therapy market with introduction of new therapies by various key players. For
instance, in November 2017, The FDA approved Mepsevii (vestronidase alfa),
which is first authorized treatment for pediatric and adult patients suffering
from an inherited metabolic condition called mucopolysaccharidosis type VII
(MPS VII).
The gaucher disease segment held
dominant position in the global lysosomal storage diseases therapeutics market
in 2018, accounting for 30.0% share in terms of value, followed by
Mucopolysaccaridosis and Fabry Diseases, respectively. In July 2017, The
European Medicines Agency (EMA) and the United States Food and Drug
Administration (FDA) collaborated to promote the use of innovative approaches
in the development of medicines for Gaucher disease. Such strategic
collaboration of regulatory bodies for the development of novel innovative drugs
is expected to boost the growth of segment over forecast period.
Market Trends
In Latin America, there are
considerable variations in requirements and time period for approval of orphan
drugs across the countries. For instance, in Brazil, the average time for
approval of orphan drug application by ANVISA is 13 months while in Mexico
average time taken for orphan drug approval is as low as 5 months.
Browse Research
Report: https://www.coherentmarketinsights.com/market-insight/lysosomal-storage-diseases-therapeutics-market-3651
In Europe, the EU funds over 100
collaborative research projects to address various aspects of rare diseases
including LSDs. For instance, the EU offers funding to a European Consortium
for Lysosomal Disorders (EUCLYD) – a network of basic and clinical
investigators in European countries to study various aspects of LSDs.
Regulations
Latin America
Global Lysosomal Storage Diseases
Therapeutics Market: Competitive Landscape
Major players operating in the
global lysosomal storage diseases therapeutics market include, Shire plc,
Pfizer, Inc., Sanofi, BioMarin Pharmaceutical Inc., Actelion Ltd., Raptor Pharmaceutical
Corp., Protalix Biotherapeutics Inc., Quest Diagnostics, and Amicus
Therapeutics, Inc.
Global Lysosomal Storage Diseases
Therapeutics Market: Key Developments
March 2020: Amicus Therapeutics,
Inc. started the company’s Global Research and Gene Therapy Center of
Excellence in uCity Square in Philadelphia to expand its portfolio of rare
disease gene therapy programs
February 2020: Lysogene, a gene
therapy platform company, received Fast Track Designation from the U.S. Food
and Drug Administration for its LYS-SAF302 program for the treatment of
mucopolysaccharidosis Type IIIA, a rare inherited neurodegenerative lysosomal
storage disorder
Buy-Now this research
report: https://www.coherentmarketinsights.com/insight/buy-now/3651
About
Coherent Market Insights:
Coherent
Market Insights is a prominent market research and consulting firm offering
action-ready syndicated research reports, custom market analysis, consulting
services, and competitive analysis through various recommendations related to
emerging market trends, technologies, and potential absolute dollar
opportunity.
Contact
Us:
mailto:sales@coherentmarketinsights.com
U.S.
Office:
Name: Mr. Shah
Coherent
Market Insights 1001 4th Ave,
# 3200
Seattle, WA 98154, U.S.
US : +1-206-701-6702
UK : +44-020-8133-4027
JAPAN : +050-5539-1737
No comments:
Post a Comment