In the single-arm, phase 2 FIREFLY-1 trial, tovorafenib produced an objective response (tumor shrinkage or resolution) in about 67% of children and young adults with relapsed or refractory, BRAF-altered pediatric…
Eflornithine (Iwilfin), an oral drug that blocks an enzyme cancer cells use to make polyamines needed for growth, is the first FDA-approved therapy specifically intended to reduce relapse risk in…
In the single-arm, phase 2 FIREFLY-1 trial, tovorafenib produced an objective response (tumor shrinkage or resolution) in about 67% of children and young adults with relapsed or refractory, BRAF-altered pediatric…
Eflornithine (Iwilfin), an oral drug that blocks an enzyme cancer cells use to make polyamines needed for growth, is the first FDA-approved therapy specifically intended to reduce relapse risk in…
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Last reviewed
FDA Approvals
Regulatory approval is separate from evidence certainty and from guideline position. Approval applies only to the exact indication stated in each jurisdiction record.
Patients 6 months to 25 years old with relapsed or refractory pediatric low-grade glioma harboring a BRAF fusion or rearrangement, or a BRAF V600 mutation, who had received at least one prior systemic therapy, enrolled in the single-arm, open-label FIREFLY-1 trial.
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Last reviewed
2026-08-13
Regulatory status by jurisdiction
United States (FDA)
Status
FDA-approved for the exact stated cancer/use
Indication
Patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma harboring a BRAF fusion or rearrangement, or a BRAF V600 mutation.
Adult and pediatric patients with high-risk neuroblastoma (a cancer arising from immature nerve cells, most common in young children) who showed at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy, enrolled in the single-arm Study 3b and compared against an external control group drawn from a separate NCI/Children's Oncology Group trial (Study ANBL0032).
View approval details
Last reviewed
2026-08-13
Regulatory status by jurisdiction
United States (FDA)
Status
FDA-approved for the exact stated cancer/use
Indication
Adult and pediatric patients with high-risk neuroblastoma who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy, to reduce the risk of relapse.
US Food and Drug Administration Approval Summary: Eflornithine for High-Risk Neuroblastoma After Prior Multiagent, Multimodality Therapy (Journal of Clinical Oncology). DOI 10.1200/JCO.24.00546, PMID 38917371.
CancerDiscoveries summarizes verified records in its database. Absence of a listing here does not establish that a treatment lacks regulatory authorization or access.