Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication Miglustat is a glucosylceramide synthase inhibitor indicated as monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option ( 1.1 ). FDA label
Also known as Zavesca
miglustat · 3 trials · 3 indications
Liver volume was assessed at baseline and end of treatment by magnetic resonance imaging. Imputation methods for patients with missing values at Month 24 were applied as follows: if a patient had at least 640 days of treatment with the study drug, the last observation that was not more than 2 days after the end of treatment was carried forward. If a patient had discontinued study drug before Day 640, the 'worst' within-patient value not more than 2 days after the end of treatment was used to impute the missing value.
Liver volume was assessed at baseline and end of treatment by magnetic resonance imaging. Imputation methods for patients with missing values at Month 24 were applied as follows: if a patient had at least 640 days of treatment with the study drug, the last observation that was not more than 2 days after the end of treatment was carried forward. If a patient had discontinued study drug before Day 640, the 'worst' within-patient value not more than 2 days after the end of treatment was used to impute the missing value.
| Arm | Type | Description |
|---|---|---|
| Open-label miglustat | EXPERIMENTAL | Oral administration of miglustat 100 mg t.i.d. for a period of 2 years |
| 1 | EXPERIMENTAL | miglustat |
| 2 | PLACEBO_COMPARATOR | placebo |
| Name | Type | Description |
|---|---|---|
| Miglustat | DRUG | Oral capsules containing miglustat 100 mg, administered three times daily (t.i.d.) |
| placebo | DRUG | Oral placebo capsules matching in appearance miglustat capsules given t.i.d. (three times a day) for 7 days and a single dose on Day 8 |
Inclusion Criteria: 1. Males or females aged 18 years or older 2. Type 1 Gaucher disease, diagnosed by glucocerebrosidase assay or molecular analysis of the glucocerebrosidase gene. 3. Treatment with ERT for at least 3 years, with a stable dose regimen for at least the last 6 months. 4. Clinically ...
Miglustat is being studied for cystic fibrosis, Gaucher disease type 1, and Niemann-Pick type C disease. It is an oral small molecule developed by Johnson & Johnson (ticker JNJ). In cystic fibrosis it is investigated as a Phase 2 therapy, while the Gaucher disease type 1 program has reached Phase 3.
Miglustat targets UGCG, also known as glucosylceramide synthase, and acts as an inhibitor of this enzyme. By blocking UGCG, miglustat reduces the synthesis of glucosylceramide, the substrate that accumulates in Gaucher disease and related lysosomal storage disorders. This mechanism underlies its investigation in Gaucher disease type 1 and Niemann-Pick type C disease.
Miglustat is developed by Johnson & Johnson, which trades under the ticker JNJ. The company is running clinical programs for miglustat in cystic fibrosis, Gaucher disease type 1, and Niemann-Pick type C disease.
Miglustat is in Phase 2 development for cystic fibrosis and Niemann-Pick type C disease, and in Phase 3 for Gaucher disease type 1. It is an investigational small molecule and is not approved for these indications based on the current development status.
Miglustat has been studied in three completed trials: NCT00742092 in cystic fibrosis (Phase 2, 11 patients, Belgium), NCT00517153 in Niemann-Pick type C disease (Phase 2, 29 patients), and NCT00319046 in Gaucher disease type 1 (Phase 3, 42 patients). All three trials are completed.