Overview
Miglustat, commonly marketed under the trade name Zavesca, is a drug used to treat Gaucher disease. It inhibits the enzyme glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids. It is only used for patients who cannot be treated with enzyme replacement therapy with imiglucerase. Miglustat is now the first and only approved therapy for patients with Niemann-Pick disease type C (NP-C). It has recently been approved for treatment of progressive neurological symptoms in adult and pediatric patients in the European Union, Brazil, and South Korea. Miglustat was first developed as an anti-HIV agent in the 1990s. However, clinical experience with miglustat showed that therapeutic levels of the drug could not be achieved in patients without a high incidence of adverse effect.
Indication
For the treatment of adult patients with mild to moderate type 1 (nonneuropathic) Gaucher's disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to constraints such as allergy, hypersensitivity, or poor venous access). Now approved in some countries for the treatment of progressive neurological symptoms in adult and pediatric patients with Niemann-Pick disease type C (NP-C).
Associated Conditions
- Mild, moderate Gaucher Disease, Type 1
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2023/11/07 | N/A | Recruiting | |||
2021/12/30 | Phase 1 | Completed | |||
2021/03/22 | Phase 3 | Recruiting | |||
2021/02/24 | Phase 2 | Completed | |||
2020/03/31 | N/A | AVAILABLE | |||
2019/10/24 | Phase 3 | Completed | |||
2019/04/11 | Phase 3 | Recruiting | |||
2019/04/10 | Phase 4 | Completed | Actelion | ||
2019/03/07 | N/A | AVAILABLE | |||
2019/01/30 | Phase 3 | Recruiting |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| Zydus Pharmaceuticals USA Inc. | 70710-2039 | ORAL | 100 mg in 1 1 | 3/17/2025 | |
| Actelion Pharmaceuticals US, Inc. | 66215-201 | ORAL | 100 mg in 1 1 | 4/20/2023 | |
| ANI Pharmaceuticals, Inc. | 43975-310 | ORAL | 100 mg in 1 1 | 8/31/2022 | |
| AMICUS THERAPEUTICS US, LLC | 71904-300 | ORAL | 65 mg in 1 1 | 9/1/2023 | |
| Edenbridge Pharmaceuticals LLC. | 42799-709 | ORAL | 100 mg in 1 1 | 10/11/2023 | |
| CoTherix, Inc. | 10148-201 | ORAL | 100 mg in 1 1 | 4/25/2023 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
Authorised | 3/22/2017 | ||
Authorised | 11/20/2002 | ||
Authorised | 2/18/2019 | ||
Authorised | 6/26/2023 | ||
Authorised | 11/9/2017 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No HSA approvals found for this drug. | |||||
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| Miglustat Capsules | 国药准字HJ20160635 | 化学药品 | 胶囊剂 | 12/27/2021 |
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| No PPB approvals found for this drug. | |||||
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| ZAVESCA miglustat 100 mg capsules blister pack | 122957 | Medicine | A | 10/23/2007 | |
| OPFOLDA miglustat 65 mg capsule bottle | 438811 | Medicine | A | 12/16/2024 |
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