Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication CERDELGA is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1) who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test [see Dosage and Administration (2.1) ] . FDA label
Also known as Cerdelga
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 being developed for the treatment of Gaucher's disease type I, a rare inherited lysosomal storage disorder. It is a small molecule under investigation for this rare disease indication. The drug has been studied in a Phase 3 trial that also included patients with Gaucher's disease type III.
Eliglustat GZ385660 targets UGCG, also known as glucosylceramide synthase. It acts as an inhibitor of this enzyme. By inhibiting UGCG, the drug aims to reduce the production of glucosylceramide, the substance that accumulates in Gaucher's disease.
Eliglustat GZ385660 is being developed by Sanofi, a global pharmaceutical company. Sanofi trades on the New York Stock Exchange under the ticker symbol SNY. The company is responsible for the clinical development of this investigational therapy for Gaucher's disease type I.
Eliglustat GZ385660 is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA for any indication. The Phase 3 trial for this drug has been completed, and further regulatory steps would be required before any potential approval.
Eliglustat GZ385660 has been evaluated in one completed Phase 3 clinical trial, NCT03485677. The trial enrolled 57 pediatric patients with Gaucher disease type 1 and type 3 across multiple countries. It was a controlled study assessing safety and efficacy, with or without imiglucerase, and included biomarker-selected patients.