On April 23, 2024, Day One Biopharmaceutical’s Ojemda^™ (tovorafenib) received Accelerated Approval from the U.S. Food and Drug Administration (FDA). A type II RAF (rapidly accelerated fibrosarcoma) inhibitor, it is indicated for the treatment of patients six months of age and older with relapsed or refractory pediatric low-grade glioma (pLGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. The recommended dose, based on body surface area, is 380mg/m2 orally once weekly, with a maximum of 600mg once weekly until disease progression or intolerable toxicity. The launch date and price have not yet been announced. Prescribing information is here.

At a Glance

• Brand (Generic) Name: Ojemda (tovorafenib)  
• Manufacturer: Day One Biopharmaceuticals
• Date Approved: April 24, 2024
• Indication: for the treatment of patients six months of age and older with relapsed or refractory pediatric low grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation
• Dosage Forms Available: 100mg oral tablets and 25mg/mL oral suspension
• Launch Date: Not yet available
• Estimated Cost: Not yet available
• The prevalence of brain tumors in pediatrics is around 5.5 per 100,000 with 30% of those accounting for pLGG. Therefore, approximately 1,200 children are diagnosed with pLGG each year in the U.S. They are the most common type of brain tumor in pediatric patients and start in the glial cells of the brain.
• Some gliomas have spontaneous mutations in the BRAF gene that can be targeted by therapy. In the U.S., it is estimated that 26,000 patients are living with BRAF alterations. It is estimated that 70% of patients with pLGG have BRAF alterations of which 85% are BRAF fusions and 15% are BRAF V600 mutations.
• Symptoms can vary depending on the region of the brain affected but can include seizures, muscle weakness, changes in behavior, vision problems, endocrine dysfunction, balance disturbances, breathing abnormalities, difficulty swallowing, and speech problems.
• Surgical resection of cancer can be up to 90% curative at 10 years, however, tumor location can be limiting. Those who have chronic progressive disease (around 70%) may require chemotherapy and/or radiation.
• Other FDA-approved therapies include Mekinist^® (trametinib – Novartis), an oral MEK inhibitor used in combination with Tafinlar^® (dabrafenib – Novartis) an oral BRAF inhibitor, to treat pLGG patients one year of age and older with a BRAF V600E mutation requiring systemic therapy.
• Accelerated approval was based on the FIREFLY-1 study. Patients who were treated with Ojemda showed an overall response rate (ORR) of 51% along with a medium duration of response (DOR) of 13.8 months. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
• Occurring in at least 30% of patients, the most common side effects include; rash, hair color changes, fatigue, viral infections, vomiting, headache, hemorrhage, fever, dry skin, constipation, nausea, rash, and upper respiratory tract infection.
• Upon approval, the FDA awarded Day One Biopharmaceuticals with a Rare Pediatric Disease Priority Review Voucher. Ojemda was also designated an Orphan drug and Breakthrough Therapy.