The Pediatric Brain Tumor Foundation (PBTF) is pleased to celebrate the FDA’s approval of Day One Biopharmaceuticals’ OJEMDA (tovorafenib), a new therapy for the treatment of pediatric low-grade glioma (pLGG), the most common brain tumor diagnosed in children. The FDA’s approval of OJEMDA represents a significant milestone for pLGG patients and their families. The new treatment also stands as a key breakthrough in the Pediatric Brain Tumor Foundation’s efforts to accelerate new treatment options for all children with brain tumors.

In 2007, a group of Boston-area families, shocked by the toxicity and lack of effectiveness of treatment options for their children, created the PLGA Foundation/A Kids’ Brain Tumor Cure (AKBTC), to fund research dedicated to understanding and treating pLGG. The PLGA Foundation/AKBTC, which later became the PLGA Fund at the Pediatric Brain Tumor Foundation, contributed early funding for research at Dana-Farber Cancer Institute that identified tovorafenib’s potential for treating pLGG tumors, as well as the seed funding for the preclinical and clinical trial work.

PBTF has since contributed significantly to Day One’s development of OJEMDA by bringing the patient voice to the process. The PBTF is proud to have provided essential support throughout the development of tovorafenib, and continues to contribute significantly to pLGG progress through ongoing research funding and advocacy.

“When we started on this journey, current treatment options for pLGG children were highly toxic and largely ineffective,” said Amy Weinstein, co-founder and former Executive Director of PLGA Foundation and former National Director of Research, PBTF. “Bringing together key stakeholders including families, like-minded foundations and government agencies, donors, and talented medical professionals, we set a vision to change the outcome for these children. Today marks a remarkable milestone that will save lives and change the standard of care for many patients, and it strengthens our collective resolve to fight this devastating disease.”  

“This is an exciting moment for children and families living with pLGG who previously had very limited treatment options if their disease progressed,” said Courtney Davies, president and chief executive officer of the Pediatric Brain Tumor Foundation. “The approval of OJEMDA is a testament to the power of community and industry collaboration to address a critical, unmet need for children whose day-to-day living and long-term health outcomes are significantly impacted by pLGG. The potential benefit that a new treatment option provides children living with this disease and their families is crucial. There is so much to celebrate here.”   

“This moment represents a major proof of concept for the work of the PLGA Fund at PBTF – and a breakthrough moment of hope for pLGG patients and their families,” said Jeffrey Gelfand, Chair & Treasurer of the Pediatric Brain Tumor Foundation’s Board of Directors. “My sincere thanks to the families in Boston who funded this early research over a decade ago, and I’m thankful that families who need OJEMDA will soon have access to it. This news demonstrates what is possible, and it motivates us to continue changing the status quo for children and survivors. Through expanded funding of pediatric brain tumor research, direct family support, and patient advocacy initiatives, we are seeking better outcomes for all patient families, every day.”

For more information on OJEMDA, view Day One’s announcement on the treatment’s FDA approval here: https://ir.dayonebio.com/news-releases/news-release-details/day-ones-ojemdatm-tovorafenib-receives-us-fda-accelerated

Those affected by a child’s brain tumor diagnosis should email patientadvocacy@curethekids.org or call 800-253-6530 to connect with the Pediatric Brain Tumor Foundation’s community of support. 

About the Pediatric Brain Tumor Foundation

Since 1991, the Pediatric Brain Tumor Foundation has led the way in funding research into pediatric brain tumors, supporting families affected by this disease, and advocating for policies that help patients, survivors, and their loved ones. Dedicated wholly to addressing this rare disease and guided by the experiences of patients, survivors, their parents, and siblings, the Pediatric Brain Tumor Foundation is the only organization to meet families’ needs along every step of their cancer journey.

The largest patient advocacy funder of pediatric brain tumor research, the foundation also funds and advocates for innovative projects that lead to vital discoveries, new clinical trials, and better treatments – all bringing us closer to a cure. We’re able to do that because of people and partners committed to supporting families and ending childhood brain cancer. Learn more at curethekids.org.

Media Contact: Amanda de Castroadecastro@groupgordon.com212 784 5724