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Trade names | Ojemda |
Other names | BIIB-024, MLN2480, AMG 2112819, DAY101, TAK-580 |
AHFS/Drugs.com | Monograph |
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Routes of administration | By mouth |
Drug class | Antineoplastic |
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Chemical and physical data | |
Formula | C17H12Cl2F3N7O2S |
Molar mass | 506.29 g·mol−1 |
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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]