drug · small molecule
miglustat
Marketedupdated 3mo agoby Biocodex🔗 ChEMBL
Approvedcommunity read0 votes
Small molecule drug with a maximum clinical stage of Approval (across all indications), with 5 approved and 9 investigational indications.
targets
Ceramide glucosyltransferase
all catalysts(0)
nothing on the calendar yet
Trials studying miglustat
- —A Global Prospective Observational Registry of Patients With Pompe Disease
- phase3A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
- phase3Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation
- phase3ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
- phase3A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
- phase2An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease
- phase4Safety and Efficacy of Miglustat in Chinese NPC Patients
- phase2Miglustat in Niemann-Pick Type C Disease
Patent cliff
source: FDA Orange Bookfirst barrier falls
Sep 28, 2026
39 days
Earliest of all listed patents + FDA exclusivities. Generic / biosimilar entry typically only becomes possible AFTER both patent and exclusivity barriers expire.
patents (10)
- Sep 16, 2038method-of-use (U-3726)US 12246062
- Aug 12, 2037method-of-use (U-3726)US 10857212
- Dec 29, 2036formulationUS 11278601
- Dec 29, 2036method-of-use (U-3726)US 12414985
- Sep 30, 2035compositionUS 10208299
- Sep 30, 2035method-of-use (U-3726)US 10961522
- Sep 30, 2035method-of-use (U-3726)US 11753632
- Mar 7, 2033method-of-use (U-3726)US 10512677
- Mar 7, 2033method-of-use (U-3726)US 11278599
- Mar 7, 2033method-of-use (U-3726)US 12419937
FDA exclusivities (1)
- Sep 28, 2026NPNP39d
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