Opfolda
(Miglustat)Phase 3Small molecule
Johnson & JohnsonOpfolda is a small molecule that works as a Glucosylceramide Synthase Inhibitor.[2] It is being investigated across 5 indications, with its most advanced program in Phase 3.[1]
Opfolda is one of 87 small molecule programs targeting Spastic paraplegia. It is currently in Phase 3.
5
Indications
18
Total Trials
3
Active Trials
86
Competitors
2
FDA Applications
NDA/BLA/ANDA regulatory filings
10
Patents
Pipeline
Status by Indication[3][1]
Indication
Competition
Competitive Landscape
386 competitors in Cystic Fibrosis
#DrugCompanyPhaseTrials
1Opfolda
Johnson & JohnsonPhase 3
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Preclinical
Actimmune
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Preclinical
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Amitiza
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Preclinical
Advil
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Preclinical
Tums
OtsukaPreclinical
Capromorelin
UUniversity of Aarhus
Preclinical
Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.
Timeline
Expected Milestones
Clinical
Trials by Indication[1]
Pompe Disease3 trials
Cystic Fibrosis3 trials
Gaucher Disease2 trials
Immunogenicity2 trials
6 more indications
GM2 gangliosidosis1 trial
Regulatory
FDA Approval Timeline[4]
Jul 31, 2003
ZAVESCA
NDA NDA021348
Sep 28, 2023
OPFOLDA
NDA NDA215211
Sources
References
- [1]
- [2]
- [3]Open Targets Platform — Drug–disease associations for Miglustat. EMBL-EBI & Wellcome Sanger Institute.
- [4]
- [5]
- [6]
Details
Drug Info
Quick Facts
- Mechanism of Action
- Glucosylceramide Synthase Inhibitor[2]
- Modality
- Small molecule[2]
- Highest Phase
- Phase 3[1]
- Company