Cerdelga (eliglustat tartrate) / Sanofi - LARVOL DELTA

Clinical Experience With Eliglustat in Children With Gaucher Disease Type 1. (PubMed, Am J Med Genet A) - No abstract available

Journal • Gaucher Disease • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Pediatrics • Rare Diseases • Type 1 Gaucher Disease

Multi-complex pharmacophore mapping for the repurposing of FDA drugs as HTLV-1 protease inhibitors: Toward novel anti-HTLV-1 therapeutics. (PubMed, Biochem Biophys Res Commun) - "Among them, the FDA-approved drug DB_FDA_1647 (Eliglustat) emerged as the most promising candidate, exhibiting favorable binding affinity, stable intermolecular interactions, and consistent structural stability throughout the molecular dynamics simulations. These findings highlight the potential of multi-complex pharmacophore mapping combined with drug repurposing as an effective strategy for developing novel anti-HTLV-1 therapeutics."

Journal • Hematological Malignancies • Leukemia • Oncology

Cumulative Antigen Suppression Reduces Clonal Plasma Cell Evolution in Gaucher Disease. (PubMed, Am J Hematol) - "Cumulative therapy progressively suppressed circulating LysoGL1, with eliglustat achieving substantially deeper reduction than enzyme replacement therapy alone. Propensity score-weighted analyses were directionally concordant (IPTW HR 0.19; 95% CI 0.11-0.30; p < 0.001). These findings establish GD as a tractable model of antigen-driven oncogenesis-and provide a precedent for investigating whether therapeutic reduction of a defined antigenic stimulus can modify oncogenic trajectory in other chronic inflammatory states."

Journal • Gaucher Disease • Genetic Disorders • Hematological Malignancies • Metabolic Disorders • Monoclonal Gammopathy • Multiple Myeloma • Oncology

TARGETING UGCG SENSITIZES AML CELLS TO VENETOCLAX THROUGH RAB32-MEDIATED ENDOPLASMIC RETICULUM-MITOCHONDRIA COMMUNICATION (EHA 2026) - "Results The combination of the UGCG inhibitor Eliglustat and Venetoclax reduced cell viability and induced apoptosis in AML cells. Interestingly, combinatory treatment activated RAB32, which led to mitochondrial fission through ER-mitochondria communication and DRP1 activation. These findings demonstrate that targeting UGCG in combination with Venetoclax is an alternative combinatory strategy to treat AML, and provide insights for ceramide- mediated cell death in anti-cancer therapies."

IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Metabolic Disorders • Solid Tumor • HSPA5 • RAB32

Modulation of tumor glycosylation to enhance antibody uptake and therapeutic efficacy (SNMMI 2026) - "PD-L1 PET imaging revealed 1.8-fold increased 64Cu-labeled avelumab uptake in mice treated with eliglustat compared to controls (Fig...Therapeutic studies combining eliglustat with anti-PD-L1 antibody (atezolizumab) and T-DXd demonstrated reduced tumor volume compared with control groups (Fig... This study demonstrates that pharmacological modulation of tumor glycosylation can be optimized to enhance antibody tumor accumulation and therapeutic efficacy. PET imaging shows that targeting N-glycans and glycosphingolipids increases anti-PD-L1 antibody uptake, supporting glycan-targeting strategies as a promising approach to improve antibody-based cancer therapies."

Clinical • IO biomarker • Oncology

Modulation of tumor glycosylation to enhance antibody uptake and therapeutic efficacy (SNMMI 2026) - "PD-L1 PET imaging revealed 1.8-fold increased 64Cu-labeled avelumab uptake in mice treated with eliglustat compared to controls (Fig...Therapeutic studies combining eliglustat with anti-PD-L1 antibody (atezolizumab) and T-DXd demonstrated reduced tumor volume compared with control groups (Fig... This study demonstrates that pharmacological modulation of tumor glycosylation can be optimized to enhance antibody tumor accumulation and therapeutic efficacy. PET imaging shows that targeting N-glycans and glycosphingolipids increases anti-PD-L1 antibody uptake, supporting glycan-targeting strategies as a promising approach to improve antibody-based cancer therapies."

Clinical • IO biomarker • Oncology

Efficacy and safety of eliglustat tartrate in adults with Gaucher disease type 1 (PubMed, Zhonghua Yi Xue Za Zhi) - "One patient reported mild nausea and anorexia, which resolved spontaneously within 2 d without other adverse events. Eliglustat tartrate demonstrates favorable efficacy and safety in these 5 adult GD1patients with EMs."

Journal • Anorexia • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

GLTP deficiency cause nEDD via disrupted epidermal glucosylceramide transport (SID 2026) - "Treatment of GLTP-deficient keratinocytes with the GlcCer synthase inhibitor eliglustat (100 µM) restored autophagic flux and significantly upregulated filaggrin mRNA expression by 7.03-fold (p < 0.0001) and loricrin by 3.4-fold (p < 0.0001). The findings establish GLTP as a novel causative gene for nEDD and unravel its indispensable role in maintaining epidermal lipid homeostasis. Pharmacological inhibition of GlcCer synthesis represents a promising therapeutic approach for treating the disorder."

Late-breaking abstract • FLG • GLTP • LORICRIN

Inhibition of glycosphingolipid synthesis overcomes the steric hindrance of CD30 N-glycans to augment CD30-targeted immunotherapeutic efficacy. (PubMed, Cell Mol Immunol) - "Furthermore, the addition of eliglustat also enhanced the tumor-killing activity of brentuximab vedotin (BV), a CD30-directed antibody-drug conjugate, both in vitro and in vivo. This glycoimmunotherapy paradigm represents a clinically actionable approach to overcome glycan-mediated immune evasion and enhance therapeutic efficacy in CD30-positive lymphomas."

Journal • Classical Hodgkin Lymphoma • Hematological Malignancies • Hodgkin Lymphoma • Lymphoma • Oncology • TNFRSF8

Real-World Effectiveness and Safety of Eliglustat in Adult Patients with Gaucher Disease Type 1: A Multicenter Retrospective Study in China. (PubMed, J Clin Med) - "No serious AE or treatment discontinuation occurred. In routine clinical practice in China, eliglustat was associated with rapid substantial reductions in plasma lyso-Gb1, early improvements in hematologic and visceral parameters, and favorable short-term tolerability in adults with GD1."

Clinical • Journal • Real-world evidence • Retrospective data • Gaucher Disease • Genetic Disorders • Hematological Disorders • Metabolic Disorders • Thrombocytopenia • Type 1 Gaucher Disease

Repurposing Gaucher disease therapy for Saposin C deficiency: Proof-of-concept with eliglustat. (PubMed, Mol Genet Metab) - "A 47-year-old patient with PSAP mutations causing Sap C deficiency who presented with features similar to those seen in GD, has received Eliglustat over the course of 9 years, demonstrating an improvement in her hepatosplenomegaly, haematological parameters, biomarkers and bone density, providing proof-of-concept that Eliglustat can be of benefit when the GCase cofactor is deficient. However, no improvement was observed in the patient's seizure activity where future brain penetrant molecules may be of benefit."

Journal • Review • CNS Disorders • Epilepsy • Gaucher Disease • Genetic Disorders • Hematological Disorders • Metabolic Disorders • GBA1 • PSAP

Targeting UGCG sensitizes AML cells to venetoclax through RAB32-mediated endoplasmic reticulum-mitochondria communication. (PubMed, Cell Rep) - "Here, we found that the inhibition of UGCG genetically or with its inhibitor eliglustat efficiently suppressed growth and promoted apoptosis in AML cells. Interestingly, combinatory treatment activated RAB32, which led to mitochondrial fission through ER-mitochondria communication and DRP1 activation. These findings demonstrate that targeting UGCG in combination with venetoclax is an alternative combinatory strategy to treat AML and provide insights into ceramide-mediated cell death in anti-cancer therapies."

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Solid Tumor • HSPA5 • RAB32

Eliglustat prevents acute kidney injury caused by Shiga toxin 2 in lethal and sublethal rat models of hemolytic uremic syndrome. (PubMed, Front Pharmacol) - "The effectiveness of EG treatment depended on the dose and the pretreatment time. The oral treatment with EG could be a therapeutic strategy to prevent the action of Stx2 and the development of acute kidney injury in diarrhea-associated HUS patients."

Journal • Preclinical • Acute Kidney Injury • Atypical Hemolytic Uremic Syndrome • Glomerulonephritis • Nephrology • Renal Disease • AQP1 • LCN2

Asymmetric Total Syntheses of Eliglustat and C2-epi-Eliglustat. (PubMed, J Org Chem) - "It is worth noting that the structure of dibenzyl acetals plays a key role in the stereochemical outcome of some of these reactions, as supported by theoretical calculations. Overall, this strategy enables the independent manipulation of the amino and hydroxy functional groups and favors the isolation of enantiomerically pure products."

Journal

GSL Synthetase Inhibitor Plus Immune Checkpoint Inhibitor and/or Regorafenib in Previously Treated pMMR/MSS CRC. (clinicaltrials.gov) - P2 | N=120 | Recruiting | Sponsor: Chinese PLA General Hospital | Not yet recruiting ➔ Recruiting | Trial completion date: Aug 2027 ➔ Mar 2029 | Trial primary completion date: Aug 2026 ➔ Mar 2027

Checkpoint inhibition • Enrollment open • IO biomarker • pMMR • Trial completion date • Trial primary completion date • Colorectal Cancer • Oncology • Solid Tumor

Adverse Event Profile Differences Between Eliglustat and Miglustat: A Pharmacovigilance Study using the U.S. Food and Drug Administration Adverse Event Reporting System. (PubMed, Drug Res (Stuttg)) - "Notably, male patients treated with eliglustat have the significantly higher incidence of weight increase and dry skin. Female patients treated with miglustat have the significantly higher incidence of dysphagia and cognitive disorder.In the clinical administration of eliglustat and miglustat, clinicians need to monitor the effects of adverse events varied by gender and to pay more attention to new adverse event signals."

Adverse events • Journal • Atopic Dermatitis • CNS Disorders • Cognitive Disorders • Dermatology • Developmental Disorders • Dyspepsia • Gastrointestinal Disorder • Immunology • Vitiligo

ELIKIDS: Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 (clinicaltrials.gov) - P3 | N=57 | Completed | Sponsor: Sanofi | Active, not recruiting ➔ Completed

Trial completion • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Pediatrics • Type 1 Gaucher Disease • GBA

GSL Synthetase Inhibitor Eliglustat Combined With CD30 Target Immunotherapy for the Treatment of of CD30+ Lymphoma (clinicaltrials.gov) - P1/2 | N=40 | Recruiting | Sponsor: Chinese PLA General Hospital | Not yet recruiting ➔ Recruiting | Initiation date: Sep 2025 ➔ Dec 2025

Enrollment open • IO biomarker • Trial initiation date • Hematological Malignancies • Lymphoma • Oncology

Cytokine-driven glycosphingolipid metabolism modulates endoplasmic reticulum calcium homeostasis in primary human renal mesangial cells. (PubMed, Front Immunol) - "Pharmacological inhibition of GSL synthesis with eliglustat significantly reduced HexCers levels and restored ER Ca2+ stores, but did not impact cytokine-induced cytokine/chemokine secretion or cell viability/proliferation. Together, these data suggest that elevated GSL synthesis modulates cytokine-induced ER Ca2+ dysregulation in mesangial cells and may play a role in the pathogenesis of lupus nephritis."

Journal • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Metabolic Disorders • Nephrology • IFNA1 • IFNG • IL1B • TNFA

Phenotypic spectrum and treatment outcomes in Russian gaucher disease patients: Real-world experience with biosimilar imiglucerase (ASH 2025) - "Therapy: 321 patients received pathogenetic treatment: enzyme replacement therapy (ERT): 92% (imiglucerase-biosimilar [Russia]: 69%, velaglucerase: 30%, taliglucerase: 1%) substrate reduction therapy (eliglustat): 8% Outcomes: after 7 years of ERT, anemia persisted in 6% and severe thrombocytopenia (platelets 93% achieving hematologic stability on long-term therapy."

Clinical • Real-world • Real-world evidence • Gaucher Disease • Gene Therapies • Genetic Disorders • Hematological Disorders • Leukopenia • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • Thrombocytopenia

Real-world outcomes with eliglustat for gaucher disease type 1 in China: A multicenter retrospective study (ASH 2025) - "Eliglustat showed significant real-world effectiveness and a favorable safety profile in Chinese GD1 patients, suggesting its potential as a first-line treatment in resource-limited settings where ERT access is challenging. While limited by small sample size and short follow-up, these findings align with global data and provide critical insights into GD1 management in China. Future long-term, prospective studies are warranted to validate these results and further explore Eliglustat's role in addressing unmet needs in this population."

Real-world • Real-world evidence • Retrospective data • Gaucher Disease • Genetic Disorders • Hematological Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • Thrombocytopenia • Type 1 Gaucher Disease

Exploring the hub gene CERS6 as a therapeutic target in type 1 diabetes through a bioinformatics and network analyst approach. (PubMed, Sci Rep) - "Methotrexate, eliglustat, myriocin and statins were identified as potential drugs for CERS6. Overall, these findings provide valuable insights that could pave the way for new experimental strategies in T1DM therapy."

Journal • Diabetes • Immunology • Metabolic Disorders • Type 1 Diabetes Mellitus

Bone involvement in Gaucher disease: Data from a North African registry. (PubMed, Reumatol Clin (Engl Ed)) - "We presented descriptive data on BI derived from the Tunisian national Gaucher disease registry. This manifestation was common in our cohort. The limited size and heterogeneity of the treated subgroups precluded robust statistical comparisons. A major challenge in our setting is the delayed initiation of specific therapies, primarily due to late diagnosis and limited access to treatment."

Journal • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Musculoskeletal Pain • Oncology • Osteoporosis • Pain • Rheumatology

ELIKIDS: baseline characteristics from the eliglustat substrate reduction therapy trial in children with Gaucher disease type 1 or type 3 (SSIEM 2023) - No abstract available

Clinical • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

ELIKIDS: baseline characteristics from the eliglustat substrate reduction therapy trial in children with Gaucher disease type 1 or type 3 (SSIEM 2023) - No abstract available

Clinical • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease