FDA Approves FoundationOne CDx as Companion Diagnostic for Tovorafenib in Pediatric Low-Grade Glioma
FoundationOne CDx is now FDA-approved as the first companion diagnostic for tovorafenib, enabling targeted treatment for relapsed/refractory pediatric low-grade glioma with BRAF mutations or rearrangements.
- The FDA granted approval to FoundationOne CDx as the first companion diagnostic for tovorafenib (Ojemda) for patients with relapsed or refractory pediatric low-grade glioma (pLGG) with BRAF mutations or rearrangements.
- This next-generation sequencing test analyzes 324 genes and critical genomic signatures, providing oncologists with precise data to guide therapy decisions while reducing reliance on traditional, aggressive treatments.
- Tovorafenib was granted accelerated approval by the FDA for the treatment of relapsed/refractory BRAF-altered pLGG in April 2024.
The FDA has approved the FoundationOne CDx to be used as a companion diagnostic for tovorafenib for the treatment of patients 6 months and older with relapsed/refractory pLGG harboring a BRAF fusion, rearrangement, or BRAF V600 mutation.
Tovorafenib was previously granted accelerated approval by the FDA for the treatment of relapsed or refractory BRAF-altered pLGG, the most common brain tumor diagnosed in children, in April 2024.2 The approval was supported by results from the phase 2 FIREFLY-1 trial where patients aged 6 months to 25 years with relapsed or progressive pLGG and a known activating BRAF alteration were administered tovorafenib once weekly as monotherapy. Here, the overall response rate was 67%, which met the study’s primary end point, the median duration of response was 16.6 months, and the median time to response was 3.0 months.3
Notably, FoundationOne CDx is the first and only companion diagnostic approved for tovorafenib.1
“Foundation Medicine is proud to partner with Day One to help healthcare providers connect pediatric patients and families with this treatment option,” said Mia Levy, MD, PhD, chief medical officer at Foundation Medicine, in a press release. “Our high-quality tissue-based companion diagnostic test is uniquely capable of detecting both BRAF V600 mutations and fusions which enables providers to gain the complete genomic picture of their patient’s tumor and guide treatment decision making.”
Human brain activity: ©See Less - stock.adobe.com
FoundationOne CDx is a next-generation sequencing-based in vitro diagnostic device that works to detect substitutions, insertion and deletion alterations, copy number alterations, and specific gene rearrangements in 324 genes. It identifies genomic signatures such as microsatellite instability and tumor mutational burden from DNA isolated from formalin-fixed, paraffin-embedded tumor tissue specimens.
As a companion diagnostic, FoundationOne CDx matches patients to targeted therapies that are approved for their specific tumor profiles. While the test can guide treatment decisions, it does not guarantee that a patient will be matched to a therapy, nor does a negative result rule out the presence of actionable alterations. Some patients may require additional biopsies to achieve a complete genomic profile.
“Historically, pediatric patients with pLGG have faced overwhelming side effects, both near- and long-term, from aggressive treatments like chemotherapy and radiation,” said David Arons, president and chief executive officer at the National Brain Tumor Society, in the press release. “We are thrilled to see that there are now additional treatment options available for these children, as well as an FDA-approved companion diagnostic test to help identify more patients who may benefit from Day One’s therapy.”
REFERENCES:
U.S. Food and Drug Administration approves FoundationOne®CDx as a companion diagnostic for OJEMDA™ (tovorafenib) to treat the most common form of childhood brain tumor in pediatric patients. News release. Foundation Medicine, Inc. January 17, 2025. Accessed January 17, 2025. https://tinyurl.com/bde6p2jy
FDA grants accelerated approval to tovorafenib for patients with relapsed or refractory BRAF-altered pediatric low-grade glioma. US FDA. April 23, 2024. Accessed January 17, 2025. https://tinyurl.com/4ah2mbu5
Kilburn LB, Khuong-Quang DA, Hansford JR, et al. The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial. Nat Med. 2024;30(1):207-217. doi:10.1038/s41591-023-02668-y
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