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
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
Table of Contents
OVERVIEWKEY TAKEAWAYSMARKETED DRUGSPIPELINE DRUGSPROBABILITY OF SUCCESSBIBLIOGRAPHYAPPENDIX
DISEASE BACKGROUND
TREATMENT
EPIDEMIOLOGY
RECENT EVENTS AND ANALYST OPINION
KEY REGULATORY EVENTS
LICENSING AND ASSET ACQUISITION DEALS
CLINICAL TRIALS
LIST OF FIGURES
LIST OF TABLES