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Opfolda · pharmadog

pharmadog
drugs
Opfolda

drug · small molecule

patient view →

miglustat

Marketedupdated 2mo ago

by Biocodex🔗 ChEMBL

Approved

community 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

News(0)

no news has mentioned this drug yet

brand

Opfolda

trade names

Miglustat dipharma, Miglustat gen.orph, Opfolda, Yargesa, Zavesca

generic

miglustat

MOA

Ceramide glucosyltransferase inhibitor

indication

Batten Disease

phase

marketed

Data sources

ClinicalTrials.gov · OpenTargets

last refreshed just now

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Patent cliff

source: FDA Orange Book

first barrier falls

Sep 28, 2026

83 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, 2026NPNP83d

Trials studying miglustat

—A Global Prospective Observational Registry of Patients With Pompe Diseaserecruitingn=500
phase3A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18recruitingn=36
phase3Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutationcompletedn=16
phase3ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPDactive not recruitingn=21
phase3A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Diseasecompletedn=125
phase2An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Diseasecompletedn=6
phase4Safety and Efficacy of Miglustat in Chinese NPC Patientscompletedn=17
phase2Miglustat in Niemann-Pick Type C Diseasecompletedn=29

more trials →