Last reviewed · How we verify
Niemann-Pick disease, type C
Approved treatments
- Zavesca · AstraZeneca
Zavesca works by inhibiting the enzyme glucosylceramide synthase, which is involved in the production of a toxic lipid that accumulates in cells of patients with certain lysosomal storage disorders.
Competitive intelligence
For the full treatment-landscape CI brief — marketed leaders side-by-side, pipeline by phase, recent regulatory actions, sponsor landscape:
- Niemann-Pick disease, type C patent landscape — cliff calendar, originator estates, attackable patents
- Niemann-Pick disease, type C treatment landscape brief
- Niemann-Pick disease, type C treatment updates RSS
Frequently asked questions about Niemann-Pick disease, type C
What drugs treat Niemann-Pick disease, type C?
Tracked treatments include Zavesca.
What is the treatment for Niemann-Pick disease, type C?
Standard treatment for Niemann-Pick disease, type C includes Zavesca. See the treatment landscape page for first-line, second-line and salvage options.
Related
- Treatment landscape: Niemann-Pick disease, type C patent cliff · Niemann-Pick disease, type C CI report
- Treatment: Zavesca · AstraZeneca
- All diseases: Browse the disease index · Disease landscape hub
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing