First Systemic Therapy for Pediatric BRAF-mutated Low-grade Glioma
The FDA approved the BRAF inhibitor tovorafenib for pediatric patients with certain Genetic alterations.
The U.S. Food and Drug Administration (FDA) has approved tovorafenib (Ojemda) for the treatment of pediatric patients aged 6 months or older with relapsed or treatment-refractory low-grade glioma (LGG) that has a fusion or rearrangement of the BRAF gene or a BRAF V600 mutation.
Tovorafenib is a targeted therapy that inhibits several kinases in the RAF family, including BRAF. When mutated, these kinases can promote uncontrolled cell proliferation and cancer growth. Tovorafenib can cross the blood-brain barrier, which prevents many medications from reaching brain tumors.
This is the first FDA approval for tovorafenib, which is the first systemic therapy approved for BRAF-mutated pediatric LGG.
The approval was based on data from the multicenter, open-label, single-arm phase II FIREFLY-1 clinical trial. Researchers enrolled 76 patients with relapsed or refractory pediatric LGG whose cancer progressed after at least one prior systemic therapy and did not harbor other known cancer-causing mutations; patients were also excluded if they had neurofibromatosis type 1.
All patients were treated with tovorafenib, with an overall response rate of 51%. Responses to tovorafenib persisted for a median of 13.8 months.
The recommended dose of tovorafenib is 380 mg per m2 of body surface area once weekly as a tablet or oral suspension, not to exceed 600 mg per week. Treatment should continue until disease progression or unacceptable toxicity.
Gliomas are the most common type of brain and spinal cord tumor diagnosed in children and adolescents. According to federal statistics, an estimated three out of every 100,000 children are diagnosed with a brain or nervous system cancer and 0.6 out of every 100,000 children die of such cancers every year.
The FDA rendered its decision on April 23, 2024.
