Drug · Small Molecule
Tovorafenib
CI-DRUG-00000599Explore in graph →NCIt C106254 CHEMBL3348923 CIViC Approved
Regulatory
Approvals (7)
Each record names the authority, jurisdiction, indication text and status. A drug approved in one jurisdiction for one indication is not 'approved' in general.
- Source
- openFDA — Drugs@FDA applications and drug labels (SPL)
- Dataset
- Drugs@FDA via openFDA
- Version
- drugsfda-2026-09-11
- Retrieved
- Sep 14, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Evidence
- regulatory
- License
- CC0 1.0 Universal (public domain; https://open.fda.gov/license/)
- Run
- ING-OPENFDA-20260914-000001
| Jurisdiction · authority | Cancer | Indication | Status | Approval date | Source |
|---|---|---|---|---|---|
| US FDA | — | Efficacy supplement 2025-08-27 (see label) | approved | Aug 27, 2025 | openfda |
| US FDA | — | Efficacy supplement 2025-08-27 (see label) | approved | Aug 27, 2025 | openfda |
| US FDA | — | OJEMDA is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication is approved under accelerated approval based on response rate and duration of response [see Clinical Studies (14) ]. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). OJEMDA is a kinase inhibitor indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. ( 1 ) This indication is approved under accelerated approval based on response rate and duration of response [see Clinical Studies (14) ]. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).accelerated | approved | Apr 23, 2024 |
Health Canada records are DIN-level: one row per marketed product (brand, strength, form). The Drug Product Database does not publish indications, so no cancer is stated for these rows, and a cancelled or dormant DIN is the status of that one product — not a withdrawal of the molecule.
Data updated 12 hours agoSource updated unknown
Derived
Development pipeline
Most advanced stage across all cancers, then per top-level cancer reached through trial conditions or approval indications. Approval in any ingested jurisdiction outranks trial phase; counts are interventional studies.
| Scope | Stage | Max phase | Active | Recruiting | Phase 3 | Trials | Approvals | Jurisdictions | First approval | First trial |
|---|---|---|---|---|---|---|---|---|---|---|
| All cancers | Approved | Phase 3 | 7 | 4 | 1 | 13 | 7 | CA, EU, US | Apr 23, 2024 | Sep 15, 2011 |
| Leukemia | Phase2 | Phase 2 | 1 | 1 | 0 | 1 | 0 | — | — | May 29, 2026 |
| Malignant Central Nervous System Neoplasm | Phase2 | Phase 2 | 1 | 0 | 0 | 1 | 0 | — | — | 2026-11 |
| Non-Hodgkin Lymphoma | Phase2 | Phase 2 | 1 | 1 | 0 | 1 | 0 | — |
Curated evidence
Clinical evidence (2)
CIViC items in which this therapy appears, grouped by cancer context, then molecular profile. 50 items per page.
- Source
- CIViC — Clinical Interpretation of Variants in Cancer
- Dataset
- CIViC evidence items
- Version
- civic-2026-09-08
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Evidence
- expert curation
- License
- CC0 1.0
- PMID
- 37978284
- Run
- ING-CIVIC-20260908-000001
| Therapy | Cancer | Type | Level | Direction · significance | Rating (1–5) | Status | Evidence | Source |
|---|---|---|---|---|---|---|---|---|
| BRAF V600E + BRAF Fusion1 | ||||||||
| Tovorafenib | Childhood Low Grade Glioma | |||||||
Clinical trials
Trials with this intervention (14)
Most recently updated first, 50 per page.
- Source
- ClinicalTrials.gov
- Dataset
- ClinicalTrials.gov API v2 studies
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
| NCT | Title | Status | Phase | Enrollment (n) | Sponsor | Countries | Last update | Source |
|---|---|---|---|---|---|---|---|---|
| NCT07441707 | A Study to Assess a Medicine Called Tovorafenib in Japanese Children and Young Adults With Brain Tumours | active not recruiting | Phase 1 | 6 | IpsenINDUSTRY | 1 | Sep 3, 2026 | clinicaltrials |
| NCT06965114 | Testing the Combination of Anti-cancer Drugs, Tovorafenib Plus Rituximab, in Patients With Hairy Cell Leukemia | recruiting | Phase 1 / Phase 2 |