Miglustat
CAS 72599-27-0 5 FDA-DMF suppliersSmall Molecule
Overview
Miglustat is an oral drug used primarily for the treatment of type 1 Gaucher disease and Niemann-Pick disease type C. It functions by inhibiting the enzyme glucosylceramide synthase, reducing the substrate accumulation that characterizes these disorders.
API suppliers (1)
Suppliers of Miglustat backed by a verifiable FDA Type II Drug Master File. Source: FDA quarterly DMF list.
- DIPHARMA SA1 active DMF
Patent & exclusivity (US)
Per the FDA Orange Book (5 approved US products), the latest listed US patent on Miglustat expires 15 Sept 2038 and exclusivity 27 Sept 2026. Generic API sourcing typically opens up once these lapse.
Therapeutic areas
Lysosomal storage disordersGaucher diseaseNiemann-Pick disease
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