Drugs / Lucerastat
Trials 7
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 15 trials · 1 met primary | ||||||
| Phase 1 | NCT03832452 | Feb → Apr 2019 | — | Idorsia Pharmaceuticals Ltd. | Completed | Met primary |
| Phase 1 | NCT03380455 | Jan 2018 | — | Idorsia Pharmaceuticals Ltd. | Completed | No outcome recorded |
| Phase 1 | NCT02930655 | Feb 2015 → Feb 2016 | Fabry disease | Idorsia Pharmaceuticals Ltd. | Completed | No outcome recorded |
| Phase 1 | NCT02944474 | Dec 2003 → May 2004 | — | Idorsia Pharmaceuticals Ltd. | Completed | No outcome recorded |
| Phase 1 | NCT02944487 | Oct → Dec 2002 | — | Idorsia Pharmaceuticals Ltd. | Completed | No outcome recorded |
| Phase 32 trials · 1 missed | ||||||
| Phase 3 | NCT03737214 | Dec 2018 → Aug 2029 expected | Fabry disease | Idorsia Pharmaceuticals Ltd. | Active not recruiting | No outcome recorded |
| Phase 3 | NCT03425539 | Jun 2018 → Aug 2021 | Fabry disease | Idorsia Pharmaceuticals Ltd. | Completed | Missed primary |
News releases announcing trial results or a regulatory action · 5
| Date | Issuer | Release |
|---|---|---|
| 2026-02-06 | Idorsia Pharmaceuticals Ltd. | Results Clear route to registration positions lucerastat as the potential first oral therapy for all patients with Fabry disease idorsia.com ↗
The results indicate that long-term treatment with lucerastat monotherapy was associated with low-to-no levels of kidney Gb3 inclusions. |
| 2026-01-30 | Idorsia Pharmaceuticals Ltd. | Results Idorsia to present long-term lucerastat data and kidney biopsy results at WORLDSymposium idorsia.com ↗
Data from up to 42 months of treatment reinforce lucerastat’s potential as a first-in-class oral substrate reduction therapy addressing key unmet needs across the Fabry population |
| 2026-01-12 | Idorsia Pharmaceuticals Ltd. | Results Nature Communications reports promising effect of Idorsia’s lucerastat on kidney function in Fabry disease idorsia.com ↗
Idorsia Ltd (SIX: IDIA) announces the publication of results from the pivotal Phase 3 MODIFY study and its open-label extension (OLE) evaluating lucerastat, an oral substrate reduction therapy, in adults with Fabry disease. |
| 2021-12-13 | Idorsia Pharmaceuticals Ltd. | Results Idorsia to further characterize lucerastat for the treatment of Fabry disease by continuing the open-label extension of the Phase 3 MODIFY study idorsia.com ↗
Lucerastat demonstrated a substantial reduction in levels of the Fabry-disease biomarker plasma Gb3 after 6 months of treatment. |
| 2021-10-11 | Idorsia Pharmaceuticals Ltd. | Results Idorsia announces the results of MODIFY, a Phase 3 study of lucerastat in Fabry disease idorsia.com ↗
Idorsia Ltd (SIX: IDIA) today announced that MODIFY, the Phase 3 study to investigate the effect of lucerastat, as an oral substrate reduction therapy for the treatment of adult patients with Fabry disease, did not meet the primary endpoint. |
All press releases naming this drug 6 releases
Evidence & citations 8 cited values
Every value below carries the sentence it was read from. 9 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Lucerastat | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03737214 ↗ |
| Known as | ACT-434964 | ChEMBL registry synonym — accepted as the source's own label CHEMBL1086997 ↗ |
| Known as | N-butyldeoxygalactonojirimycin | ChEMBL registry synonym — accepted as the source's own label CHEMBL1086997 ↗ |
| Known as | N-n-butyl deoxygalactonojirimycin | ChEMBL registry synonym — accepted as the source's own label CHEMBL1086997 ↗ |
| Action | Inhibit | “Lucerastat, an inhibitor of glucosylceramide synthase” PMID 28088251 ↗ Jan 20171“Ceramide glucosyltransferase inhibitor” CHEMBL1086997 ↗ |
| Modality | Small molecule | “Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders” PMID 28088251 ↗ Jan 2017 |
| Route | Oral | “Ten subjects with Fabry Disease received 1000 mg of oral lucerastat twice daily for 12 weeks” NCT02930655 ↗ |
| Target | UGCG | “Ceramide glucosyltransferase inhibitor” CHEMBL1086997 ↗ |