By Bill Thomas | May 15, 2024
Not long ago, the Pediatric Cancer Research Foundation hosted a special guest blog written by Dr. Samuel C. Blackman, co-founder and head of research and development at Day One Biopharmaceuticals. In his article, Dr. Blackman described his company’s development of tovorafenib as a potential treatment for pediatric Low-Grade Glioma (pLGG) brain tumors.
In an important update, the U.S. Food and Drug Administration recently granted accelerated approval to the weekly pill, which will be marketed as Ojemda. It is indicated for patients 6 months of age and older with relapsed or refractory pediatric Low-Grade Glioma (pLGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.
This represents the first FDA approval of a systemic therapy for the treatment of patients with pediatric Low-Grade Glioma (pLGG) with BRAF rearrangements, including fusions.
The most common brain tumor diagnosed in children, pLGG is a chronic and relentless cancer that can devastate children and their families. BRAF is the most commonly altered gene in pLGG, with up to 75% of children having a BRAF alteration. Until now, there have been no medicines approved for patients with pLGG driven by BRAF fusions.
Ojemda’s advancement, as described in Dr. Blackman’s blog, proves that it is possible to develop new medicines for pediatric cancers if done with deliberation and urgency. It also reinforces the power of collaboration that is so vital to our work and embraced by all of us here at the Pediatric Cancer Research Foundation.
Anyone interested in learning more about OJEMDA can read the press release issued by Day One Biopharmaceuticals, the FDA press release or the paper about its key clinical trial published in Nature Medicine. To stay up to date with all the latest news shaping the future of pediatric cancer treatment, don’t forget to follow the Pediatric Cancer Research Foundation Profectus blog!
