Novartis on Friday announced the U.S. Food and Drug Administration (FDA) granted approval for Tafinlar (dabrafenib) + Mekinist (trametinib) for the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy.
According to a press statement, the FDA also approved liquid formulations of Tafinlar and Mekinist, marking the first time a BRAF/MEK inhibitor has been developed in a formulation suitable for patients as young as one year of age. These approvals make Tafinlar + Mekinist the first and only approved combination targeted therapy to treat pediatric patients with BRAF V600E LGG, it added.
“Pediatric cancer research is vital to uncover new treatment methods for a population. Developing targeted therapies based on the unique genetic features of a patient’s tumor is the future of pediatric cancer care,” said Dr. Eric Bouffet, MD, FRCPC, Principal Investigator of the TADPOLE clinical trial and Associate Scientist Emeritus at The Hospital for Sick Children (SickKids) in a statement.
According to the company, the safety profile of Tafinlar + Mekinist observed in this study was consistent with the known safety profile in other approved indications. The most common adverse reactions (>=15%) were pyrexia (68%), rash (51%), headache (47%), vomiting (34%), musculoskeletal pain (34%), fatigue (33%), diarrhea (29%), dry skin (26%), nausea (25%), hemorrhage (25%), abdominal pain (25%), dermatitis acneiform (22%), dizziness (15%), upper respiratory tract infection (15%) and weight increased (15%).
These data were highlighted as part of an official press briefing and oral presentation at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting.
“This new indication for Tafinlar + 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. 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,” said Reshema Kemps-Polanco, Executive Vice President, US Oncology at Novartis in a statement.
LGG is the most common pediatric brain cancer. BRAF V600 mutations are present in 15-20% of pediatric LGGs and are associated with poor survival outcomes and less favorable response to chemotherapy4. BRAF mutations have been identified as drivers of cancer growth across a wide range of solid tumors, and often have limited treatment options, it claimed.