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Market Spotlight: Lysosomal Storage Disorders

  • ID: 5148348
  • Report
  • May 2021
  • Region: Global
  • 64 pages
  • Datamonitor Healthcare
This Market Spotlight report covers the Lysosomal Storage Disorders market, comprising epidemiology, key marketed and pipeline drugs, recent events and analyst opinion, key regulatory events, licensing and asset deals, probability of success, and clinical trials.

Key Takeaways
  • Among the 50+ individual lysosomal storage disorders (LSDs), mucopolysaccharidosis (MPS) (all subtypes combined) is the most common disorder, followed by Gaucher disease, metachromatic leukodystrophy, and Fabry, Krabbe, and Pompe diseases.
  • According to the epidemiology literature, combined MPS prevalence ranges from 1.0 to 4.8 per 100,000, Gaucher disease prevalence ranges from 0.25 to 11.6 per 100,000, Fabry disease prevalence ranges from 0.12 to 0.85 per 100,000, Krabbe disease prevalence ranges from 0.14 to 1.35 per 100,000, and Pompe disease prevalence ranges from 0.10 to 2.66 per 100,000 live births.
  • The approved drugs in the LSDs space focus on a wide variety of targets. The majority of the approved drugs are administered via the intravenous route, with the remainder being oral, topical, and intracerebral/cerebroventricular formulations.
  • Therapies in development for LSDs focus on a wide variety of targets. The majority of drugs in development for LSDs are administered via the intravenous route, with the remainder being oral, intracerebral/cerebroventricular, intrathecal, intraarticular, subcutaneous, and intramuscular formulations.
  • The overall likelihood of approval of a Phase I inborn errors of metabolism asset is 38.1%, and the average probability a drug advances from Phase III is 78.6%. Drugs, on average, take 10.5 years from Phase I to approval, compared to 9.6 years in the overall metabolic space
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OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND
  • Definition
  • Subtypes
  • Risk factors
  • Diagnosis
TREATMENT
  • Enzyme replacement therapy
  • Hematopoietic stem cell transplantation
  • Substrate reduction therapy
  • Chaperone therapy
  • Gene therapy
EPIDEMIOLOGY
  • Lysosomal storage disorders epidemiology
MARKETED DRUGS

PIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION
  • AT-GAA for Pompe Disease (February 11, 2021)
  • AXO-AAV-GM1 for GM1 Gangliosidosis (December 15, 2020)
  • IB1001 for Niemann-Pick Disease (September 30, 2020)
  • Avalglucosidase Alfa for Pompe Disease (June 16, 2020)
  • Olipudase Alfa for Niemann-Pick Disease (January 30, 2020)
  • ELX-02 for Cystinosis (January 14, 2020)
  • Miplyffa for Niemann-Pick Disease (January 3, 2020)
  • Pegunigalsidase alfa for Fabry's Disease (October 17, 2019)
KEY REGULATORY EVENTS
  • Latest Japan Approvals Include World-First For Pabinafusp
  • Latest EU Filings
  • Sanofi Nabs Priority Review For New Pompe Drug
  • Positive On Brineura
  • Gene Therapies Dominate New Crop Of EU PRIME Recipients
  • Cancer, Rare Disease Drugs Among New China Approvals
  • NDAs And More: Recent Submissions For FDA Approval
  • China Approves More Drugs For Cancer, Rare Diseases
PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS
  • Japan’s SDP Taps Gain For Lysosomal Disorders Drug Discovery
  • Chiesi Licenses Blood-Brain Barrier Technology From Bioasis
CLINICAL TRIALS
  • Amicus Pompe Disease Combo’s Approvability In Question As Key Trial Misses Mark
BIBLIOGRAPHY

APPENDIX

LIST OF FIGURES
Figure 1: Relative frequencies of lysosomal storage disorders
Figure 2: IB1001 for Niemann-Pick Disease (September 30, 2020): Phase II - 201
Figure 3: Avalglucosidase Alfa for Pompe Disease (June 16, 2020): Phase III - COMET
Figure 4: Olipudase Alfa for Niemann-Pick Disease (January 30, 2020): Phase II/III - ASCEND
Figure 5: ELX-02 for Cystinosis (January 14, 2020): Phase II - McGill University (Canada)
Figure 6: Pegunigalsidase alfa for Fabry's Disease (October 17, 2019): Phase III - BRIDGE
Figure 7: Probability of success in the inborn errors of metabolism pipeline

LIST OF TABLES
Table 1: Prevalence proportions of mucopolysaccharidosis subtypes
Table 2: Prevalence proportions of Gaucher disease
Table 3: Prevalence proportions of Fabry disease
Table 4: Prevalence proportions of Krabbe disease
Table 5: Prevalence proportions of Pompe disease
Table 6: Marketed drugs for lysosomal storage disorders
Table 7: Pipeline drugs for lysosomal storage disorders in the US
Table 8: AT-GAA for Pompe Disease (February 11, 2021)
Table 9: AXO-AAV-GM1 for GM1 Gangliosidosis (December 15, 2020)
Table 10: IB1001 for Niemann-Pick Disease (September 30, 2020)
Table 11: Avalglucosidase Alfa for Pompe Disease (June 16, 2020)
Table 12: Olipudase Alfa for Niemann-Pick Disease (January 30, 2020)
Table 13: ELX-02 for Cystinosis (January 14, 2020)
Table 14: Miplyffa for Niemann-Pick Disease (January 3, 2020)
Table 15: Pegunigalsidase alfa for Fabry's Disease (October 17, 2019)
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