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: Eastern America
Company Founding Year: 2008
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: China, Japan, Taiwan, United States
Active Clinical Trial Count: 5
Recent & Upcoming Milestones
- Vorasidenib, a potential breakthrough therapy for idh-mutant glioma, granted priority review with a PDUFA date of 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 |
Oligodendroglioma|Astrocytoma |
2026-01-31 |
50% |
2025-06-04 |
Primary Completion Date|Primary Endpoints|Treatments |
NCT06780930 |
CL3-95032-016 | P3 |
Active, not recruiting |
Glioma |
2025-10-02 |
6% |
2026-01-10 |
Primary Completion Date|Primary Endpoints|Start Date |
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 |
jRCT2061250027 |
jRCT2061250027 | P3 |
Recruiting |
Glioma |
2026-02-28 |
Recent News Events
Date |
Type |
Title |
|---|---|---|
|
01/20/2026 |
News Article |
Royalty Pharma to Announce Fourth Quarter and Full Year 2025 Financial Results on February 11, 2026 |
|
01/12/2026 |
News Article |
Royalty Pharma to Highlight Accomplishments and Provide Business Update at 44th Annual J.P. Morgan Healthcare Conference |
|
01/09/2026 |
News Article |
Royalty Pharma Announces Dividend Increase |
|
12/29/2025 |
News Article |
Royalty Pharma Acquires Remaining Royalty Interest in Roche's Evrysdi for $240 Million and Potential Milestones |
|
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? |
|
02/29/2024 |
PubMed |
A Promising Breakthrough: The Potential of VORASIDENIB in the Treatment of Low-grade Glioma. |