Gaucher Disease Type I
Also known as: Type I Gaucher Disease / Gaucher Disease, Type I / Gaucher Disease, Type 1 / Type 1 Gaucher Disease / Gaucher Disease Type 1 / Gaucher's disease type I / Type 1 Gaucher's disease / Chronic non-neuropathic Gaucher's disease (disorder)
Drug | Drug Name | Drug Description |
---|---|---|
DB09039 | Eliglustat | A glucosylceramide synthase used to treat type 1 Gaucher disease in patients who are CYP2D6 extensive, intermediate, or poor metabolizers. |
DB00053 | Imiglucerase | A form of recombinant human beta-glucocerebrosidase enzyme used to replace the deficient endogenous enzyme in the treatment of Gaucher disease. |
DB08876 | Taliglucerase alfa | A hydrolytic lysosomal glucocerebroside specific enzyme, used for long term enzyme replacement therapy in patients with Type 1 Gaucher disease. |
DB06720 | Velaglucerase alfa | An enzyme replacement therapy used for the long-term treatment of for pediatric and adult patients with type 1 Gaucher disease. |
Drug | Drug Name | Target | Type |
---|---|---|---|
DB09039 | Eliglustat | Ceramide glucosyltransferase | target |
DB09039 | Eliglustat | Cytochrome P450 2D6 | enzyme |
DB09039 | Eliglustat | Cytochrome P450 3A4 | enzyme |
DB09039 | Eliglustat | P-glycoprotein 1 | transporter |
DB00053 | Imiglucerase | Glucocerebroside | target |
DB08876 | Taliglucerase alfa | Glucocerebroside | target |
DB06720 | Velaglucerase alfa | Glucosylceramidase | target |
Drug | Drug Name | Phase | Status | Count |
---|---|---|---|---|
DB09039 | Eliglustat | 1 | Completed | 1 |
DB06742 | Ambroxol | 1 / 2 | Suspended | 1 |
DB06151 | Acetylcysteine | 2 | Recruiting | 1 |
DB04545 | Afegostat | 2 | Completed | 3 |
DB06742 | Ambroxol | 2 | Completed | 1 |
DB05025 | Arimoclomol | 2 | Terminated | 1 |
DB05715 | Eliglustat | 2 | Completed | 1 |
DB09039 | Eliglustat | 2 | Completed | 1 |
DB00053 | Imiglucerase | 2 | Active Not Recruiting | 1 |
DB14966 | Venglustat | 2 | Active Not Recruiting | 1 |
DB09039 | Eliglustat | 3 | Active Not Recruiting | 1 |
DB05715 | Eliglustat | 3 | Completed | 2 |
DB09039 | Eliglustat | 3 | Completed | 2 |
DB00053 | Imiglucerase | 3 | Active Not Recruiting | 1 |
DB00053 | Imiglucerase | 3 | Completed | 3 |
DB00419 | Miglustat | 3 | Completed | 1 |
DB05543 | Velaglucerase alfa | 3 | Completed | 3 |
DB06720 | Velaglucerase alfa | 3 | Completed | 3 |
DB00053 | Imiglucerase | 4 | Completed | 2 |
DB08876 | Taliglucerase alfa | 4 | Withdrawn | 1 |
DB06720 | Velaglucerase alfa | 4 | Completed | 1 |
DB06720 | Velaglucerase alfa | 4 | Withdrawn | 1 |
DB08876 | Taliglucerase alfa | Not Available | Completed | 1 |
DB05543 | Velaglucerase alfa | Not Available | Approved for Marketing | 1 |
DB06720 | Velaglucerase alfa | Not Available | Approved for Marketing | 1 |