Ojemda FDA Approval History
Last updated by Judith Stewart, BPharm on April 26, 2024.
FDA Approved: Yes (First approved April 23, 2024)
Brand name: Ojemda
Generic name: tovorafenib
Dosage form: Tablets and Oral Suspension
Company: Day One Biopharmaceuticals, Inc.
Treatment for: Low-Grade Glioma
Ojemda (tovorafenib) is a type II pan-RAF kinase inhibitor for the treatment of pediatric low-grade glioma.
- Ojemda is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (pLGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.
This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). - Pediatric low-grade glioma (pLGG) is the most common brain tumor diagnosed in children, accounting for 30-50 percent of all central nervous system tumors. It can lead to vision loss and motor dysfunction, among other disease-related morbidities. Up to 75 percent of children with pLGG have a BRAF alteration. In children with BRAF-altered pLGG, approximately 80 percent have BRAF fusions or rearrangements, while the remaining 20 percent have a V600 mutation.
- Ojemda is an inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases. It works by targeting a key enzyme in the Mitogen-Activated Protein Kinase (MAPK) signaling pathway, which is involved in gene expression as well as the growth and survival of cells.
- FDA accelerated approval of Ojemda was based on data from the FIREFLY-1 trial of 137 patients across two study arms. Of the 76 patients in arm 1 of the study, the best overall response rate was 51%, which included 28% partial responses and 11% minor responses.
- Ojemda tablets and oral suspension are administered once weekly, with or without food.
- Warnings and precautions associated with Ojemda include major hemorrhagic events, skin reactions including photosensitivity, hepatotoxicity, reductions in growth velocity, embryo-fetal toxicity, and neurofibromatosis type 1 associated tumors.
- Common adverse reactions (≥30%) include rash, hair color changes, fatigue, viral infection, vomiting, headache, hemorrhage, pyrexia, dry skin, constipation, nausea, dermatitis acneiform, and upper respiratory tract infection.
Common Grade 3 or 4 laboratory abnormalities (≥2%) 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.
Development timeline for Ojemda
Further information
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