Product Description
Vorasidenib, an investigational, oral, selective, brain-penetrant inhibitor of mutant IDH1 and IDH2 enzymes, is currently being evaluated in the registration-enabling Phase 3 INDIGO study as a potential treatment for patients with residual or recurrent Grade 2 non-enhancing glioma. (Sourced from: https://investor.agios.com/news-releases/news-release-details/agios-presents-updated-data-phase-1-dose-escalation-study)
Mechanisms of Action: IDH Inhibitor
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: *
Approval Status: Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Agios
Company Location: CAMBRIDGE MA 02139
Company CEO: Jacqualyn A. Fouse
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: China, Japan, United States
Active Clinical Trial Count: 5
Recent & Upcoming Milestones
- Agios to receive $1.1B upon FDA approval of vorasidenib, with PDUFA action date set for August 20, 2024.
Highest Development Phases
Phase 3: Glioma
Phase 1: Astrocytoma|Healthy Volunteers|Oligodendroglioma
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT05484622 |
CL1-95032-005 | P1 |
Recruiting |
Astrocytoma|Oligodendroglioma |
2026-01-31 |
50% |
2025-06-04 |
Primary Completion Date|Primary Endpoints|Treatments |
jRCT2061250027 |
jRCT2061250027 | P3 |
Recruiting |
Glioma |
2026-02-28 |
|||
NCT05609994 |
ViCToRy | P1 |
Recruiting |
Glioma |
2027-08-01 |
50% |
2025-07-08 |
Primary Endpoints|Start Date|Treatments|Trial Status |
NCT05843708 |
PKH-95032-009 | P1 |
Completed |
Healthy Volunteers |
2023-07-28 |
23% |
2025-02-15 |
Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments|Trial Status |
NCT06780930 |
CL3-95032-016 | P3 |
Recruiting |
Glioma |
2026-06-30 |
34% |
2025-01-18 |
Primary Endpoints|Treatments |
Recent News Events
Date |
Type |
Title |
|---|---|---|
|
12/08/2025 |
News Article |
The Galien Foundation Honors 2025 Prix Galien Bridges Awards Recipients |
|
12/08/2025 |
News Article |
Servier's Voranigo® (vorasidenib) Receives Prix Galien Awards Across Multiple Regions |
|
12/04/2025 |
News Article |
Royalty Pharma and Denali Therapeutics Announce $275 Million Royalty Funding Agreement |
|
12/04/2025 |
News Article |
Denali Therapeutics and Royalty Pharma Announce $275 Million Royalty Funding Agreement |
|
02/29/2024 |
PubMed |
A Promising Breakthrough: The Potential of VORASIDENIB in the Treatment of Low-grade Glioma. |
|
05/13/2024 |
PubMed |
Mutant IDH inhibitors induce lineage differentiation in IDH-mutant oligodendroglioma. |
|
01/09/2024 |
PubMed |
Vorasidenib in IDH mutant WHO grade 2 gliomas: time to stop sitting on the fence? |