Last update 10 Mar 2026

Tovorafenib

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
AMG-2112819, BIIB-024, DAY101
+ [3]
Action
inhibitors
Mechanism
PDK1 inhibitors(3-phosphoinositide-dependent protein kinase 1 inhibitors), Raf kinase inhibitors(RAF serine/threonine protein kinase inhibitors)
Originator Organization
Drug Highest PhaseApproved
RegulationPriority Review (United States), Breakthrough Therapy (United States), Accelerated Approval (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Orphan Drug (Japan)
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Structure/Sequence

Molecular FormulaC17H12Cl2F3N7O2S
InChIKeyVWMJHAFYPMOMGF-ZCFIWIBFSA-N
CAS Registry1096708-71-2

External Link

KEGGWikiATCDrug Bank
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R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
BRAF fusion or rearrangement postive low-grade glioma
United States
23 Apr 2024
BRAF V600 mutation low-grade glioma
United States
23 Apr 2024
Low grade glioma
United States
23 Apr 2024
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
NeoplasmsNDA/BLA
Canada
01 Oct 2025
Malignant glioma of brainPhase 3
Sweden
28 Nov 2022
Hairy Cell LeukemiaPhase 2
United States
27 Jun 2026
Refractory Langerhans Cell HistiocytosisPhase 2
United States
28 Mar 2024
Refractory Langerhans Cell HistiocytosisPhase 2
Australia
28 Mar 2024
Refractory Langerhans Cell HistiocytosisPhase 2
Canada
28 Mar 2024
Advanced Malignant Solid NeoplasmPhase 2
United States
-23 Sep 2021
Advanced Malignant Solid NeoplasmPhase 2
Australia
-23 Sep 2021
Advanced Malignant Solid NeoplasmPhase 2
Canada
-23 Sep 2021
Advanced Malignant Solid NeoplasmPhase 2
Denmark
-23 Sep 2021
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 2
137
prvunqrmmo(owiohslkap) = pcebwpovwj gfdygxzlvm (ijteczzknd )
Positive
24 Nov 2025
(Exploratory analyses:entered a treatment-free observation period)
dfembbyube(qxxwhyhxzi) = eetdhifbum yvqvdpbpel (uxtzyhsrmm )
Phase 2
23
(Melanoma Cohort)
ulhighfyhz = koupwcsraa wlpolqemhs (zfflmqflkb, qlystbqlxw - fwapxzrcrd)
-
02 Oct 2025
(Tissue Agnostic Cohort)
ulhighfyhz = hdorlwwysj wlpolqemhs (zfflmqflkb, exwasgidiz - digfciwbnk)
Phase 2
137
hflmldeijb(rbbtwtjlpi) = uouefvavdk iikpwzsozn (dhnuybuqhw )
-
30 May 2025
Phase 1/2
167
exfxkkxgtj(anowdkycaj) = fyqxwhpjic mixfliceuf (udmtvdzqcb, 0.4 - 2.2)
-
30 May 2025
Phase 2
-
evcyffncoq(cozrtvrsop) = 39% ywyenfemqn (pdutoejxtx )
-
14 Sep 2024
Phase 1
Low grade glioma
MAPK pathway activated
35
Tovorafenib 530 mg/m2
hkpafaaujn(ctfoepcupn) = zelukbctww cbqxkeadmc (fxqvxomwdh )
Positive
24 May 2024
Phase 2
137
kucdpqqgll(slagpzruli) = qxuldzyqpg gpjnfhulfw (jsppegenth, 40 - 63)
Positive
23 Apr 2024
Phase 2
136
fnmhaziorn(zbxzxtlkrs) = hyibvnmucn lfsimqcrio (zeivcofnym )
Positive
10 Nov 2023
MAPK inhibitor therapy
fnmhaziorn(zbxzxtlkrs) = prpidbjayk lfsimqcrio (zeivcofnym )
Phase 2
Optic Nerve Glioma
BRAF V600E mutation
77
tmvmmkmcaj(woxvpzrwka) = pjnqsbywkm mlglfqchxm (gneerdqicj )
Positive
10 Nov 2023
Phase 2
77
yybbzzfchb(dcykcwllav) = httccnrhha jryaqqclct (tprpyboxjy )
Positive
31 May 2023
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Approval

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Regulation

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