Product Description
Eliglustat is used to treat Gaucher disease type 1 (a condition in which a certain fatty substance is not broken down normally in the body and builds up in some organs and causes liver, spleen, bone, and blood problems) in certain people. Eliglustat is in a class of medications called enzyme inhibitors. It works by preventing the body from producing the fatty substance so that less of it will build up in the body and cause symptoms. (Sourced from: https://medlineplus.gov/druginfo/meds/a618038.html)
Mechanisms of Action: GCS Inhibitor
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Approved
Approved Countries: Argentina | Australia | Austria | Belgium | Bosnia | Brazil | Canada | Chile | Colombia | Croatia | Czech | Denmark | Dominican Republic | Ecuador | Estonia | European Medicines Agency | Finland | France | Germany | Greece | Hungary | Iceland | India | Ireland | Israel | Italy | Japan | Korea | Latvia | Lithuania | Luxembourg | Netherlands | Norway | Peru | Poland | Portugal | Romania | Russia | Saudi Arabia | Serbia | Slovakia | Slovenia | Spain | Sweden | Switzerland | Taiwan | Turkey | United Kingdom | United States
Approved Indications: Gaucher Disease
Known Adverse Events: Abdominal Pain | Back Pain | Headache | Pain Unspecified | Diarrhea
Company: Sanofi
Company Location: PARIS I0 75008
Company CEO: Paul Hudson
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Argentina, Canada, France, Italy, Japan, Russia, Spain, Sweden, Turkey, United Kingdom
Active Clinical Trial Count: 4
Highest Development Phases
Phase 3: Gaucher Disease
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| JapicCTI-195058 | P3 |
Active |
Gaucher Disease |
2025-11-30 |
|
| EFC13738 | P3 |
Unknown Status |
Gaucher Disease |
2025-11-20 |
|
| ELIKIDS | P3 |
Active, not recruiting |
Gaucher Disease |
2025-11-20 |
95% |
| ELIKIDS | P3 |
Active, not recruiting |
Gaucher Disease |
2025-11-20 |
95% |
| ELIKIDS | P3 |
Active, not recruiting |
Gaucher Disease |
2025-10-09 |