Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Eliglustat GZ385660 · 1 trial · 2 indications
Maximum concentration (Cmax) of eliglustat in plasma
Area under the plasma eliglustat concentration-time curve (AUC)
Number of adverse events in pediatric patients
| Arm | Type | Description |
|---|---|---|
| Cohort 1: Eliglustat monotherapy | EXPERIMENTAL | Eliglustat for at least two years. Cohort 1 patients that experience significant clinical decline will receive rescue treatment. Rescue Treatment Step 1: Switch from eliglustat to imiglucerase monotherapy. Rescue Treatment Step 2: Patients who after 6 months of rescue therapy with imiglucerase monotherapy do not show improvement in the parameter(s) that led to the switch from eliglustat to imiglucerase, will then receive combination therapy with eliglustat + imiglucerase. |
| Cohort 2: Eliglustat plus imiglucerase | EXPERIMENTAL | Eliglustat plus imiglucerase for three years, at the dose of enzyme replacement therapy received before enrollment. After Week 52, Cohort 2 patients will switch to eliglustat monotherapy for the remainder of the study if the desired clinical response has been achieved. |
| Name | Type | Description |
|---|---|---|
| Eliglustat GZ385660 | DRUG | Pharmaceutical form: Capsule, Liquid Route of administration: Oral |
| Imiglucerase GZ437843 | DRUG | Pharmaceutical form: Powder for solution for infusion Route of administration: Intravenous |
Inclusion criteria : * The participant were 2 to \<18 years old at the time of informed consent. * Male and female participants with a clinical diagnosis of Gaucher disease (GD) type 1 or type 3 with documented deficiency of acid beta-glucosidase activity by enzyme assay and glucocerebrosidase (GBA...
Eliglustat GZ385660 is an investigational small molecule being studied for the treatment of Gaucher's Disease Type I, a rare genetic disorder. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.
Eliglustat GZ385660 is being developed by Sanofi, a multinational pharmaceutical company listed on the stock exchange under the ticker SNY. The drug is in Phase 3 clinical development for Gaucher's Disease Type I.
Eliglustat GZ385660 is in Phase 3 clinical development. It is an investigational drug and has not been approved for marketing. One Phase 3 clinical trial has been completed, with no active trials currently ongoing.
Eliglustat GZ385660 has been studied in one completed Phase 3 clinical trial, identified as NCT03485677. This trial evaluated the safety and efficacy of eliglustat with or without imiglucerase in pediatric patients with Gaucher Disease Type 1 and Type 3, enrolling 57 participants.
Eliglustat GZ385660 is a specific designation of eliglustat, a small molecule drug being developed by Sanofi. It is being studied for Gaucher's Disease Type I and is currently in Phase 3 clinical development.