OPFOLDA (miglustat)
Opfolda is an enzyme stabilizer used for adults with late-onset Pompe disease who weigh at least 40 kg. It helps patients with this specific enzyme deficiency when their current enzyme replacement therapy is no longer providing sufficient improvement. This medication is taken in combination with Pombiliti to help manage the condition more effectively.
How OPFOLDA Works
This drug works by binding to and stabilizing the enzyme cipaglucosidase alfa-atga in the bloodstream, which prevents it from becoming inactive after infusion. Once the enzyme is transported into the lysosomes, the miglustat separates from it, allowing the enzyme to perform its function. While miglustat itself does not break down glycogen, it ensures the accompanying enzyme therapy remains stable enough to reach its target.
Details
- Status
- Prescription
- First Approved
- 2023-09-28
- Patent Cliff
- 2038
- Routes
- ORAL
- Dosage Forms
- CAPSULE
OPFOLDA Approval History
What OPFOLDA Treats
1 indicationsOPFOLDA is approved for 1 conditions since its original approval in 2023. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Pompe Disease
OPFOLDA Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to OPFOLDA
3 of 4FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
9 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT04808505 ROSSELLA | ATB200-08 | Ph 3 | recruiting | A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 |
| NCT03911505 | ATB200-04 | Ph 3 | active not recruiting | ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD |
| NCT03822013 EMTISTD | 97-01-30-36953 | Ph 3 | terminated | Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) |
| NCT03729362 PROPEL results posted | ATB200-03 | Ph 3 | completed | A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease |
| NCT03910621 | AC-056C405 | Ph 4 | completed | Safety and Efficacy of Miglustat in Chinese NPC Patients |
| NCT02030015 Syner-G results posted | Syner_G_Regimen U54NS065768, 1311M46101 | Ph 4 | terminated | Synergistic Enteral Regimen for Treatment of the Gangliosidoses |
| NCT00742092 | AC-056A202 | Ph 2 | completed | Miglustat in Cystic Fibrosis |
| NCT01760564 | 200802043M | Ph 3 | completed | Application of Miglustat in Patients With Niemann-Pick Type C |
| NCT00945347 | MIG-99 | Ph 2 | completed | Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ? |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
OPFOLDA FDA Label Details
Indications & Usage
FDA Label (PDF)OPFOLDA is indicated for the treatment of Pompe Disease.
OPFOLDA Patents & Exclusivity
Patents (10 active)
Exclusivity
Pro Intelligence Preview
Deep insights for OPFOLDA
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2038
- • 10 active patents
Trial Analysis
- • Clinical trial tracking
- • Development stage analysis
Competitive Landscape
- • 4 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.