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Lysosomal storage diseases therapeutics market | Coherent Market Insights

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Title: Lysosomal storage diseases therapeutics market | Coherent Market Insights


1
Coherent Marketing Insights
  • Market Research Business Consulting Services
  • https//www.coherentmarketinsights.com/
  • Covid-19 Impact Tracker

2
  • LYSOSOMAL STORAGE DISEASES THERAPEUTICS MARKET
    ANALYSIS
  • Lysosomal Storage Diseases Therapeutics Market,
    By Treatment (Enzyme Replacement Therapy (Stem
    Cell Therapy, Substrate Reduction Therapy,
    Others)), By Indication (Gaucher's Disease, Fabry
    Disease, Pompes Syndrome, Mucopolysaccharidosis,
    Others), By End User (Hospitals, Clinics), and By
    Region (North America Latin America Europe Asia
    Pacific Middle East Africa) - Size, Share,
    Outlook, and Opportunity Analysis, 2019 - 2027

3
  • Lysosomal storage diseases (LSDs) are a group of
    rare inherited metabolic disorders that result
    from defects in lysosomal function. The absence
    of lysosomal enzymes such as hydrolase
    responsible for breakdown of specific substrate,
    its activator or a transporter, result in
    accumulation of substrates, leading to LSDs.
    Examples of LSDs include the mucopolysaccharidoses
    , mucolipidoses, oligosaccharidoses, Pompe
    disease, gaucher disease, Fabry disease, the
    Niemann-Pick disorders, and neuronal ceroid
    lipofuscinoses.
  • 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.

4
  • Global Lysosomal Storage Diseases Therapeutics
    Market Drivers
  • Significant prevalence of LSDs is expected to
    boost growth of the global lysosomal storage
    diseases therapeutics market over the forecast
    period. For instance, according to a report
    updated by National Center for Biotechnology
    Information, in January 2020, the prevalence of
    Fabry disease in white, male populations was
    117,000 to 1117,000. Moreover, classic Fabry
    disease mutations were evident in around 122,000
    to 140,000 males and atypical presentations were
    associated with about 11000 to 13000 males and
    16000 to 140,000 females.
  • Moreover, increasing awareness regarding rare
    diseases is also expected to aid in growth of the
    market. For instance, the European Organization
    for Rare Diseases celebrates Rare Disease Day
    annually on the last day of the month of
    February.
  • North America region held dominant position in
    the global lysosomal storage diseases
    therapeutics market in 2018, accounting for 32.4
    share in terms of value, followed by Europe.

5
  • Global Lysosomal Storage Diseases Therapeutics
    Market Restraints
  • Geographic dispersal of LSDs hampers efforts to
    understand the disease, given that certain rare
    diseases are even rarer in some regions. For
    instance, birth prevalence of LDSs differ
    considerably between various countries and
    population groupsAshkenazi Jewish Ancestry have
    high prevalence of several genetic diseases,
    including some LSDs. Also, there is unusually
    high prevalence of Mucopolysaccharidosis (MPS)
    VI, GM1 gangliosidosis and fucosidosis in UAE,
    owing to ethnic isolation.
  • Moreover, high cost of enzyme replacement therapy
    for the treatment of LDSs is also expected to
    hamper growth of the market.

6
Global Lysosomal Storage Diseases Therapeutics
Market Opportunities
  • RD in LSDs is expected to offer lucrative growth
    opportunities for players in the global lysosomal
    storage diseases therapeutics market. For
    instance, in February 2020, researchers from
    BioStrategies LC, State University (U.S.),
    reported that biodistribution studies in LSD
    models provide evidence for the theory that the
    RTB-lectin transports corrective doses of enzymes
    across the bloodbrain barrier for the treatment
    of central nervous system pathologies.
  • Moreover, use of advanced technologies in the
    treatment of LSDs is also expected to aid in
    growth of the market. For instance, in November
    2019, researchers from Nacional Health Institute
    Doctor Ricardo Jorge, Portugal, reported that
    CRISPR-Cas9 has great potential for successful
    disease modelling of sphingolipidoses, an
    important group of rare diseases among LSDs.

7
Market Trends/Key Takeaways
  • The cost of therapies for the treatment of LSDs
    is high. For instance, Elaprase was regarded as
    one of the most expensive drugs by Forbes Index,
    which costs US 375,000 per year whereas
    Naglazyme costs US 365,000/patient/year.
  • In the U.S., Medicare and Medicaid programs
    provide good extent of reimbursement for LSDs.
    Around 84 of prescription drug plans covered
    under Medicare part D and part B provide
    reimbursement for orphan drugs for FDA approved
    indication. For instance, cost of Fabrazyme
    therapy reimbursed under Medicare part B is 80
    of the allowable amount for Fabrazyme, for
    beneficiaries who administer Fabrazyme at a
    physicians office or as hospital outpatient.

8
Regulations
  • North America

9
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., and Amicus Therapeutics, Inc.
  • Global Lysosomal Storage Diseases Therapeutics
    Market Key Developments
  • Major players in the market are focused on
    adopting MA strategies to expand their product
    portfolio. For instance, in January 2020, Quest
    Diagnostics acquired Blueprint Genetics to expand
    its product portfolio in genetic and rare
    diseases.
  • Major players in the market are also focused on
    adopting collaboration strategies to expand their
    portfolio. For instance, in 2019, Amicus
    Therapeutics, Inc. collaborated with the
    University of Pennsylvania, under which the
    company received disease-specific worldwide
    rights to the universitys Next Generation Gene
    Therapy Technologies from the Wilson Lab for LSDs
    and other twelve rare diseases.

10
  • Request Sample Report
  • Request Customization
  • Download PDF Brochure
  • Talk to Analyst
  • Referance https//www.coherentmarketinsights.com/
    market-insight/lysosomal-storage-diseases-therapeu
    tics-market-3651

11
About Us
  • Coherent Market Insights is a global market
    intelligence and consulting organization focused
    on assisting our plethora of clients achieve
    transformational growth by helping them make
    critical business decisions. We are headquartered
    in India, having office at global financial
    capital in the U.S. Our client base includes
    players from across all business verticals in
    over 150 countries worldwide.
  • What We ProvideCustomized Market Research
    AnalysisIndustry Analysis ServicesBusiness
    Consulting ServicesLong Term Engagement
    ModelCountry Specific Analysis

12
Contact Us
  • Mr. Shah
  • Coherent Market Insights Pvt. LTD.Coherent
    Market Insights 1001 4th Ave, 3200 Seattle, WA
    98154, U.S.Phone 1-206-701-6702
  • Email sales_at_coherentmarketinsights.com

13
Thank You
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