marked.bio

pan-RAF inhibitor · small molecule

tovorafenib

5 active trials across 5 of our biomarkers, targeting RAF (type II). 2 active approvals for a biomarker on marked.bio.

Also known as DAY101, TAK-580, MLN2480, Ojemda.

Counts only trials that select patients by one of the biomarkers on marked.bio, not every trial of this drug.

Approvals and pivotal results

Biomarker-selected approvals in the United States and the European Union, with the pivotal trial's results and side effects where we have them. Results from different trials are not directly comparable.

TherapyBiomarkerTumourUnited StatesEuropean Union
tovorafenib
FDA, 2024
relapsed or refractory, BRAF fusion/rearrangement or BRAF V600 mutation, age >=6 months AA 23/04/2024; still on FDA Ongoing accelerated approvals list. Source
EU (EMA), 2026
progressed after >=1 prior systemic therapy, BRAF fusion/rearrangement or BRAF V600 mutation, age >=6 months Conditional MA 20/04/2026. Source
OjemdaBroader label 		BRAF V600E paediatric low-grade glioma Approved 2024 accelerated Approved 2026 conditional

In the US label

FIREFLY-1 single-arm, 76 patients
Response 51% (40-63)Lasting 13.8 months

Serious side effects 45%Stopped for side effects 7%

Trial details and side effects
Patients
relapsed or refractory paediatric low-grade glioma with a BRAF alteration (NCT04775485)
Most common side effects
rash 77%, hair colour changes 76%, viral infection 55%, fatigue 55%, vomiting 50%, headache 45% (137 patients)
Notes
Accelerated approval. Includes BRAF fusions as well as V600 mutations. Ojemda US label rev. 04/2024.
Source
US label

In the EU product information

FIREFLY-1 single-arm, 76 patients
Response 53% (RAPNO-LGG); 71% of 69 (RANO-HGG) (41-64)Lasting 18.0 months (RAPNO-LGG)

Trial details and side effects
Patients
relapsed or refractory paediatric low-grade glioma with a BRAF alteration (NCT04775485)
Most common side effects
rash 77%, hair colour changes 76%, viral infection 55%, fatigue 55%, vomiting 50%, headache 45% (137 patients)
Notes
EU SmPC section 5.1 (FIREFLY-1, conditional authorisation). Response by RAPNO-LGG criteria includes minor responses. Side effects from the US label.
Source
EU product information

Biomarkers

BiomarkerActive trialsAll trialsFurthest active phase
BRAF V600E 5 6 Phase 2
FGFR2 fusion or rearrangement 1 1 Phase 2
FGFR3 alteration (mutation or fusion) 1 1 Phase 2
KRAS G12C 1 1 Phase 2
KRAS G12D 1 1 Phase 2

Sponsors

Industry sponsors leading active biomarker-selected trials of tovorafenib.

  1. Day One Biopharmaceuticals 1
  2. Ipsen 1

Trials

5 of 6 trials shown

  1. Study of Tovorafenib in High-Grade Glioma and Diffuse Intrinsic Pontine Glioma (DIPG)

    Not yet recruiting Phase 2 Nationwide Children's Hospital Starts 2027Primary completion 2030-02 NCT07206849

    tovorafenib

    KRAS/NRAS alteration (Strata B or C)

    KRAS G12CKRAS G12DBRAF V600EFGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementHigh Grade Glioma (HGG) of the Brain With BRAF Aberration, High Grade Glioma (III or IV), Diffuse Intrinsic Pontine Glioma, High Grade Glioma

  2. Testing the Combination of Anti-cancer Drugs, Tovorafenib Plus Rituximab, in Patients With Hairy Cell Leukemia

    Recruiting Phase 1/2 National Cancer Institute (NCI) Started 2026Primary completion 2030-03 NCT06965114

    cladribinerituximabtovorafenib FDA EU

    …ly confirmed diagnosis of classical hairy cell leukemia (HCL), including demonstration of BRAF V600E mutation by immunohistochemistry, molecular diagnostic testing, or polymerase chain react…

    BRAF V600EHairy Cell Leukemia, Recurrent Hairy Cell Leukemia, Refractory Hairy Cell Leukemia

  3. A Study to Evaluate Tovorafenib in Pediatric and Young Adult Participants With Relapsed or Progressive Low-Grade Glioma and Advance Solid Tumors

    Recruiting Phase 2 Day One Biopharmaceuticals Started 2021Primary completion 2027-05 NCT04775485

    tovorafenib FDA EU

    …ow-Grade Glioma Extension: a relapsed or progressive LGG with documented known activating BRAF alteration.

    BRAF V600ELow-grade Glioma, Advanced Solid Tumor

  4. A Study to Assess a Medicine Called Tovorafenib in Japanese Children and Young Adults With Brain Tumours

    Active, not recruiting Phase 1 Ipsen Started 2026Primary completion 2030-07 NCT07441707

    tovorafenib FDA EU

    …rogressive low-grade glioma with a documented known activating BRAF alteration, including BRAF V600 mutations and KIAA1549:BRAF fusions, as identified through molecular assays as routinely …

    BRAF V600ELow-grade Glioma

  5. Pilot Study of Vinblastine and Tovorafenib in Pediatric Patients With Recurrent/Progressive RAF Altered Low Grade Gliomas

    Recruiting Early Phase 1 Daniel Morgenstern Started 2024Primary completion 2027-03 NCT06381570

    tovorafenib FDA EUvinblastine

    a) Progressive/Recurrent LGG (non-NF1) with documented BRAF or CRAF alteration as identified through molecular assays as routinely performed at CLIA or other similar ce…

    BRAF V600ELow-grade Glioma