On March 16, 2023, the U.S. Food and Drug Administration approved a new treatment combination that will now be available as an option for pediatric patients 1 year of age and older with low-grade glioma (LGG, grade 1 or 2) that have a mutation called BRAF V600E. This marks the first FDA approval of a treatment option for patients of this age group with LGG that have this mutation, including gangliogliomas, pleomorphic xanthoastrocytoma (PXA), as well as certain other astrocytomas and oligodendrogliomas.
The drug combination, Dabrafenib (Tafinlar, Novartis) and trametinib (Mekinist, Novartis), has also been approved in new oral formulations for patients who cannot swallow pills, which is critical for this population.
In June 2022, the FDA approved the combination of these same oral drugs for treatment of both adult and pediatric (older than 6 years of age) patients with high- and low-grade gliomas with the BRAF V600E mutation.
“We are pleased to see the expansion of this treatment option to this vulnerable population of children ages 1-6 less than a year after approval in older patients,” said David Arons, CEO and president of the National Brain Tumor Society. “We thank Novartis, and the researchers, clinicians, patients, and families involved in these studies, for making a breakthrough possible for a community with no other options.”
NBTS strongly urges that patients and care partners ensure that BRAF V600E is among biomarkers tested for by pathology. Learn more about the importance of biomarkers and biomarker testing for patients with brain tumors.
NBTS offers a Personalized Support and Navigation service staffed by a team with deep experience navigating brain tumor treatment options and questions. Patients and families who have questions about this or other options can contact the NBTS team at firstname.lastname@example.org.