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FDA Approves Tafinlar Plus Mekinist for Pediatric Brain Tumors

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This approval is the first of its kind as a first-line systemic therapy for pediatric patients with low-grade gliomas of a specific mutation.

The Food and Drug Association (FDA) approved Tafinlar (dabrafenib) in combination with Mekinist (trametinib) as a first-line therapy for low-grade gliomas with a BRAF V600E mutation for patients aged 1 year and older. This is the first FDA approval of a systemic therapy for children with this common pediatric brain cancer, according to the agency.

The approval is based on data from the TADPOLE trial demonstrating the Tafinlar and Mekinist combination generated an overall response rate (percentage of patients whose disease shrunk from treatment) of 46.6% as well as a median progression-free survival (average time from treatment until disease worsening) of 20.1 months, as opposed to a 10.8% overall response rate and progression-free survival of 7.4 months for the current standard of care.

BRAF V600E-mutant low-grade glioma is the most common pediatric brain tumor, with an average five-year survival rate of 6.8% and an estimated average length of survival after diagnosis of only eight months, according to the National Brain Tumor Society. Prior to this approval, only five treatment options were available for this treatment-resistant tumor subtype.

“This FDA approval may offer new hope to pediatric patients living with BRAF V600E low-grade glioma,” said Dr. Roger Packer, senior vice president of the Center for Neurosciences and Behavioral Medicine at Children’s National Hospital, in the press release. “This has the potential to change the way health care providers treat these pediatric patients, offering a significant advancement compared to chemotherapy.”

The most common side effect for this combination was fever, followed by rash, headache, vomiting, musculoskeletal pain, fatigue, diarrhea, dry skin, nausea, hemorrhage, abdominal pain, dizziness, upper respiratory tract infection and weight gain.

The combination therapy works by blocking tumor growth signals and has previously been approved for BRAF V600 solid tumors of other cancer types, such as lung cancer and melanoma. Of note, the FDA approved liquid formulations of Tafinlar and Mekinist for patients who cannot swallow pills, providing a unique benefit to pediatric patients.

“This new indication for Tafinlar plus Mekinist is a potential new standard of care treatment option for young patients with this form of brain cancer with a BRAF V600E mutation, in formulations specifically designed for them,” said Reshema Kemps-Polanco, executive vice president of US Oncology at Novartis, the manufacturer of both drugs. “We are thankful for the families, including children and adolescents, that participated in the clinical trial that led to this approval and whose bravery has led to a new hope for children living with this serious brain cancer.”

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