Tovorafenib

Tovorafenib

Clinical data
Trade names	Ojemda
Other names	BIIB-024, MLN2480, AMG 2112819, DAY101, TAK-580
AHFS/Drugs.com	Monograph
License data	US DailyMed: Tovorafenib
Routes of administration	By mouth
Drug class	Antineoplastic
ATC code	None
Legal status
Legal status	US: ℞-only[1]
Identifiers
IUPAC name 6-amino-5-chloro-N-[(1R)-1-[5-[[[5- chloro-4-(trifluoromethyl)-2-pyridinyl]amino]carbonyl]-2-thiazolyl]ethyl]-4-pyrimidinecarboxamide
CAS Number	1096708-71-2
PubChem CID	25161177
DrugBank	DB15266
ChemSpider	28637796
UNII	ZN90E4027M
KEGG	D12291
ChEBI	CHEBI:167672
ChEMBL	ChEMBL3348923
PDB ligand	QOP (PDBe, RCSB PDB)
Chemical and physical data
Formula	C17H12Cl2F3N7O2S
Molar mass	506.29 g·mol−1
InChI InChI=1S/C17H12Cl2F3N7O2S/c1-6(28-15(31)12-11(19)13(23)27-5-26-12)16-25-4-9(32-16)14(30)29-10-2-7(17(20,21)22)8(18)3-24-10/h2-6H,1H3,(H,28,31)(H2,23,26,27)(H,24,29,30)/t6-/m1/s1 Key:VWMJHAFYPMOMGF-ZCFIWIBFSA-N

Tovorafenib, sold under the brand name Ojemda, is a medication used for the treatment of glioma.^[1][2] It is a kinase inhibitor.^[1]

The most common adverse reactions include rash, hair color changes, fatigue, viral infection, vomiting, headache, hemorrhage, pyrexia, dry skin, constipation, nausea, dermatitis acneiform, and upper respiratory tract infection.^[3] The most common grade 3 or 4 laboratory abnormalities include decreased phosphate, decreased hemoglobin, increased creatinine phosphokinase, increased alanine aminotransferase, decreased albumin, decreased lymphocytes, decreased leukocytes, increased aspartate aminotransferase, decreased potassium, and decreased sodium.^[3]

Tovorafenib was approved for medical use in the United States in April 2024,^[1][3][4][5] and is the first approval of a systemic therapy for the treatment of people with pediatric low-grade glioma with BRAF rearrangements, including fusions.^[3][6]

Tovorafenib is indicated for the treatment of people six months of age and older with relapsed or refractory pediatric low-grade glioma harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.^[1][3][2]

Efficacy was evaluated in 76 participants enrolled in FIREFLY-1 (NCT04775485), a multicenter, open-label, single-arm trial in participants with relapsed or refractory pediatric low-grade glioma harboring an activating BRAF alteration detected by a local laboratory who had received at least one line of prior systemic therapy.^[3] Participants were required to have documented evidence of radiographic progression and at least one measurable lesion.^[3] Participants with tumors harboring additional activating molecular alterations (e.g., IDH1/2 mutations, FGFR mutations) or with a known or suspected diagnosis of neurofibromatosis type 1 were excluded.^[3] Participants received tovorafenib based on body surface area (range: 290 to 476 mg/m2, up to a maximum dose of 600 mg) once weekly until they experienced disease progression or unacceptable toxicity.^[3] The US Food and Drug Administration (FDA) granted the application for tovorafenib priority review, breakthrough therapy, and orphan drug designations.^[3]

Tovorafenib was approved for medical use in the United States in April 2024.^{[1][3][4][7][8]}

Tovorafenib is the international nonproprietary name.^[9]

• "Tovorafenib". NCI Drug Dictionary.
• "Tovorafenib (Code C106254)". NCI Thesaurus.
• Clinical trial number NCT04775485 for "A Study to Evaluate DAY101 in Pediatric and Young Adult Patients With Relapsed or Progressive Low-Grade Glioma and Advance Solid Tumors (FIREFLY-1)" at ClinicalTrials.gov