The U.S. Food and Drug Administration granted approval on Tuesday for Tovorafnib (now OJEMDA) for patients 6 months and older for the treatment of pediatric low-grade glioma.
The Team Jack Foundation invested in this drug in 2017 as a phase 1 study at Dana-Farber Cancer Institute. The drug was purchased by Day One Biopharmaceuticals to continue to accelerate the drug to market in 2020.
The Team Jack Foundation’s mission is to support groundbreaking research initiatives and raise national awareness for the disease. Brain cancer remains one of the most challenging types of cancer to treat in pediatric patients. A press release stated that though relatively rare compared to other forms of cancer, brain tumors are a leading cause of cancer-related deaths in children. This underscores the critical need for continued research, awareness, and innovative treatment options to improve outcomes for children diagnosed with this devastating disease.
“Today proves that with the generosity and support of many individuals and organizations in Nebraska and beyond, we can make a meaningful difference in the lives of children fighting brain cancer,” Kylie Dockter, Team Jack Foundation Executive Director, said in a press release. "This is a huge win for children fighting low-grade gliomas. We won’t stop fighting for kids with brain cancer until there is a cure.”
In 2017, the Team Jack Foundation committed $300,000 for a phase I study of the drug that would benefit children with low-grade gliomas. Low-grade gliomas account for approximately two-thirds of all childhood brain tumors and are the most common central nervous system tumors in children. Due to the initial outcomes of this study, in 2019 Team Jack committed an additional $500,000 for both TAK 580 and another phase I trial called MEK 162. Most low-grade gliomas arise due to defects in a single signaling pathway called RAS/RAF. Our investment allowed physicians to investigate this new molecularly targeted drug that turns off this pathway. This results in a more effective, less toxic treatment for children with this type of brain tumor.
OJEMDA is for patients with relapsed or refractory pLGG harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication is approved under accelerated approval based on response rate and duration of response. With the approval, Day One received a rare pediatric disease priority review voucher from the FDA.