Lysosomal storage diseases therapeutics market | Coherent Market Insights

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• 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

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• 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.

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• 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.

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• 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.

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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.

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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.

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Regulations

• North America

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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.

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