Last reviewed · How we verify
Zavesca (Miglustat)
Zavesca (Miglustat) is a Small molecule drug developed by Children's National Research Institute. It is currently in Phase 3 development.
Zavesca (Miglustat) is a small molecule inhibitor of ceramide glucosyltransferase, a type of enzyme. It is used to treat conditions such as GM2 Gangliosidoses, Tay-Sachs, Sandhoff Disease, and Late-onset Pompe Disease.
-
Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas).
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | Zavesca (Miglustat) |
|---|---|
| Sponsor | Children's National Research Institute |
| Modality | Small molecule |
| Phase | Phase 3 |
Approved indications
Common side effects
Key clinical trials
- A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease (PHASE2)
- A Global Prospective Observational Registry of Patients With Pompe Disease
- A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) (PHASE3)
- A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 (PHASE3)
- Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation (PHASE2, PHASE3)
- ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD (PHASE3)
- First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 (PHASE1, PHASE2)
- Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Zavesca (Miglustat) CI brief — competitive landscape report
- Zavesca (Miglustat) updates RSS · CI watch RSS
- Children's National Research Institute portfolio CI
Frequently asked questions about Zavesca (Miglustat)
What is Zavesca (Miglustat)?
Who makes Zavesca (Miglustat)?
What development phase is Zavesca (Miglustat) in?
Related
- Manufacturer: Children's National Research Institute — full pipeline
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing