Gaucher Disease Treatments

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Brand Name

Cerdelga

Generic Name
Eliglustat
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FDA approval date: September 03, 2014
Classification: Glucosylceramide Synthase Inhibitor
Form: Capsule

What is Cerdelga (Eliglustat)?

CERDELGA is indicated for the long-term treatment of adult patients with Gaucher disease type 1 who are CYP2D6 extensive metabolizers , intermediate metabolizers , or poor metabolizers as detected by an FDA-cleared test [see Dosage and Administration.

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Related Clinical Trials

GSL Synthetase Inhibitor in Combination With Immune Checkpoint Inhibitor and/or Regorafenib for Patients With Advanced/Metastatic pMMR/MSS Colorectal Cancer:an Open-Label, Randomized,Phase II Study
GSL Synthetase Inhibitor in Combination With Immune Checkpoint Inhibitor and/or Regorafenib for Patients With Advanced/Metastatic pMMR/MSS Colorectal Cancer:an Open-Label, Randomized,Phase II Study
Enrollment Status: Recruiting
Publish Date: February 11, 2026
Intervention Type: Drug
Study Phase: Phase 2

Summary: In this single-center,open-label, randomized, phase II study, the efficacy and feasibility of GSL synthetase inhibitor in combination with immune checkpoint inhibitor and/or regorafenib therapeutic regimen will be evaluated in patients with advanced/metastatic proficient mismatch repair/microsatellite stable (pMMR/MSS) colorectal cancer (CRC).In this clinical trial, a total of 120 eligible patient...

GSL Synthetase Inhibitor Eliglustat Combined With CD30 Target Immunotherapy for the Treatment of of CD30+ Lymphoma: an Open-Label, Randomized, Phase I/II Study
GSL Synthetase Inhibitor Eliglustat Combined With CD30 Target Immunotherapy for the Treatment of of CD30+ Lymphoma: an Open-Label, Randomized, Phase I/II Study
Enrollment Status: Recruiting
Publish Date: January 02, 2026
Intervention Type: Drug
Study Phase: Phase 1/Phase 2

Summary: Targeted therapy against the CD30 molecule has achieved some progress in CD30-positive Hodgkin lymphoma, but its efficacy remains unsatisfactory. Previous studies have demonstrated that N-glycan modifications in the extracellular domain of target proteins can disrupt immune synapse formation with CAR-T cells. Our preliminary research has shown that ablation of N-glycans on CD30 enhances the anti-t...

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Related Latest Advances

Adverse Event Profile Differences Between Eliglustat and Miglustat: A Pharmacovigilance Study using the U.S. Food and Drug Administration Adverse Event Reporting System.
Adverse Event Profile Differences Between Eliglustat and Miglustat: A Pharmacovigilance Study using the U.S. Food and Drug Administration Adverse Event Reporting System.
Journal: Drug research
Published: February 05, 2026
Real-World Effectiveness and Safety of Eliglustat in Adult Patients with Gaucher Disease Type 1: A Multicenter Retrospective Study in China.
Real-World Effectiveness and Safety of Eliglustat in Adult Patients with Gaucher Disease Type 1: A Multicenter Retrospective Study in China.
Journal: Journal of clinical medicine
Published: January 15, 2026
Repurposing Gaucher disease therapy for Saposin C deficiency: Proof-of-concept with eliglustat.
Repurposing Gaucher disease therapy for Saposin C deficiency: Proof-of-concept with eliglustat.
Journal: Molecular genetics and metabolism
Published: December 16, 2025
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