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]

Medical uses

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]

History

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]

Society and culture

Legal status

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

Names

Tovorafenib is the international nonproprietary name.[9]

References

1. "Ojemda- tovorafenib kit; Ojemda- tovorafenib tablet, film coated". DailyMed. 26 April 2024. Retrieved 14 May 2024.
2. Zhang T, Xu B, Tang F, He Z, Zhou J (November 2024). "Type II RAF inhibitor tovorafenib for the treatment of pediatric low-grade glioma". Expert Review of Clinical Pharmacology. 17 (11): 999–1008. doi:10.1080/17512433.2024.2418405. PMID 39412085.
3. "FDA grants accelerated approval to tovorafenib for patients with relapsed or refractory BRAF-altered pediatric low-grade glioma". U.S. Food and Drug Administration (FDA). 23 April 2024. Archived from the original on 23 April 2024. Retrieved 25 April 2024. This article incorporates text from this source, which is in the public domain.
4. "Novel Drug Approvals for 2024". U.S. Food and Drug Administration (FDA). 29 April 2024. Archived from the original on 30 April 2024. Retrieved 30 April 2024.
5. New Drug Therapy Approvals 2024 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2025. Archived from the original on 21 January 2025. Retrieved 21 January 2025.
6. Dhillon S (August 2024). "Tovorafenib: First Approval". Drugs. 84 (8): 985–993. doi:10.1007/s40265-024-02069-6. PMID 38967715.
7. "Cancer Accelerated Approvals". U.S. Food and Drug Administration (FDA). 1 October 2024. Retrieved 6 December 2024.
8. "Day One's Ojemda (tovorafenib) Receives US FDA Accelerated Approval for Relapsed or Refractory BRAF-altered Pediatric Low-Grade Glioma (pLGG), the Most Common Form of Childhood Brain Tumor". Day One Biopharmaceuticals (Press release). 23 April 2024. Archived from the original on 23 April 2024. Retrieved 24 April 2024.
9. World Health Organization (2022). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 88". WHO Drug Information. 36 (3). hdl:10665/363551.

External links

• "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