Miglustat
As of the edition of 20 October 2026, Miglustat is a small molecule: marketed, first approved in 2002 (EU); acts on UGCG; no US protection of its own is listed.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Marketed; latest approval 2012
- Kind
- Small molecule
- Brand name
- Zavesca
- First approved
- 20 November 2002 (EU)
- Approvals on record
- 7 in 4 regions
- Holder of the most recent approval
- Amicus Therapeutics; 7 holders in all
- Registered trials
- 29, 3 stopped early
- Disease areas
- Inherited and neonatal; Nervous system; Nutrition and metabolism; Blood and lymph; Digestive system; Immune system
US protection
No US protection of its own is listed.
The FDA registers list no drug-substance patent or exclusivity of its own that counts for this drug.
Amicus Therapeutics holds a later exclusivity on its own new formulation or combination product, ending 2035; it does not protect the original drug.
This is the US only.
Companies that need a new molecule because of this drug
2 companies on route B because of this drug.
Companies on route B for this drug
In the product2 companies are on route B because of Miglustat.
See the 2 companies on route B for this drug, with the facts behind eachRequest access. Nothing is shown until you do.
Target
| Gene | Protein | UniProt |
|---|---|---|
| UGCG | Ceramide glucosyltransferase | Q16739 |
Registered trials
| Phase | Trials | Completed | Active | Stopped early |
|---|---|---|---|---|
| Phase not stated | 5 | 1 | 1 | 0 |
| Phase 1 | 2 | 2 | 0 | 0 |
| Phase 2 | 10 | 9 | 0 | 1 |
| Phase 3 | 9 | 6 | 2 | 1 |
| Phase 4 | 3 | 2 | 0 | 1 |
| All trials | 29 |
Who has held its approvals
| Company | Role | Years |
|---|---|---|
| Johnson & Johnson | Applicant and Lead sponsor of trials | 2002 to now |
| Advanz Pharma | Acquired holder | 2002 to now |
| Amicus Therapeutics | Applicant, Later holder and Lead sponsor of trials | 2023 to now |