FDA Approves FoundationOne®CDx for OJEMDA™ in Pediatric Brain Tumor
Foundation Medicine, Inc. announced today that the U.S. Food and Drug Administration (FDA) has approved FoundationOne®CDx as a companion diagnostic for Day One Biopharmaceuticals’ OJEMDA, a type II RAF inhibitor. This approval is for the treatment of pediatric patients aged six months and older with relapsed or refractory pediatric low-grade glioma (pLGG) that harbors a BRAF fusion, rearrangement, or the BRAF V600 mutation. FoundationOne®CDx is now the first and only companion diagnostic approved for use with OJEMDA.
Pediatric low-grade glioma (pLGG) is the most common brain tumor diagnosed in children, with a significant proportion of these tumors harboring BRAF mutations. BRAF alterations are detected in up to 75% of pLGG cases, with BRAF fusions accounting for approximately 80% of these alterations. Prior to recent developments, there were no FDA-approved treatments specifically targeting tumors with BRAF fusions. This left a large unmet need in the pediatric oncology space, as BRAF fusions represent a substantial portion of the BRAF-altered patient population. The FDA approval of FoundationOne®CDx as a companion diagnostic for Day One Biopharmaceuticals’ OJEMDA, a type II RAF inhibitor, marks a significant step forward in addressing this gap.
FoundationOne®CDx is a comprehensive tissue-based diagnostic test that can detect both BRAF V600 mutations and BRAF fusions, providing clinicians with a complete genomic profile of a patient’s tumor. This capability enables healthcare providers to make more informed treatment decisions and ensure that patients receive therapies tailored to the specific genetic characteristics of their tumors. The approval of FoundationOne®CDx as a companion diagnostic for OJEMDA offers pediatric patients with relapsed or refractory pLGG a new treatment option and brings hope to families dealing with this challenging disease.
“We are proud to partner with Day One to help healthcare providers connect pediatric patients and families with this treatment option,” said Mia Levy, M.D., Ph.D., Chief Medical Officer at Foundation Medicine. “Our high-quality tissue-based companion diagnostic test is uniquely capable of detecting both BRAF V600 mutations and fusions, which enables providers to gain a comprehensive genomic picture of their patient’s tumor and guide treatment decision-making.”
Foundation Medicine is a recognized leader in the field of cancer genomics. The company has sequenced more than 2,200 pediatric central nervous system (CNS) tumors, providing valuable insights into the genetic underpinnings of pediatric brain cancers. FoundationOne®CDx, an FDA-approved test, analyzes over 300 cancer-related genes in a tumor sample to identify genetic alterations that may influence treatment options. Foundation Medicine is the only company offering both tissue and blood-based comprehensive genomic profiling tests that are approved by the FDA. In addition, Foundation Medicine leads the industry with 40% of all FDA-approved companion diagnostic indications for next-generation sequencing (NGS) testing in both the U.S. and Japan.
The approval of FoundationOne®CDx as a companion diagnostic for OJEMDA represents a significant milestone in the treatment of pediatric brain tumors. Previously, pediatric patients with pLGG often faced overwhelming side effects from aggressive treatments such as chemotherapy and radiation. These treatments, while sometimes effective, come with both immediate and long-term risks to a child’s health and quality of life. The availability of OJEMDA, combined with FoundationOne®CDx as a companion diagnostic, offers a new, potentially less toxic treatment option for these patients.
David Arons, President and CEO of the National Brain Tumor Society, expressed his excitement about this advancement: “Historically, pediatric patients with pLGG have faced overwhelming side effects, both near- and long-term, from aggressive treatments like chemotherapy and radiation. 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.”
FoundationOne®CDx is a next-generation sequencing (NGS)-based in vitro diagnostic device that can detect various genetic alterations in tumor samples. It analyzes substitutions, insertions and deletions (indels), copy number alterations (CNAs), and gene rearrangements in 324 genes. The test also provides information on genomic signatures, such as microsatellite instability (MSI) and tumor mutational burden (TMB). FoundationOne®CDx is intended for prescription use only and serves as a companion diagnostic to identify patients who may benefit from certain targeted therapies. It is designed to assist healthcare providers FDA in making personalized treatment decisions based on a patient’s tumor mutation profile.
While FoundationOne®CDx is a powerful tool for precision medicine, it is important to note that a negative result does not rule out the presence of an alteration. Some patients may require additional biopsies or testing to obtain a comprehensive understanding of their tumor’s genetic makeup. The test is intended to be used by qualified healthcare professionals following established oncology guidelines for patients with solid malignant tumors.
About Foundation Medicine: Foundation Medicine is a leading provider of molecular profiling services that aim to transform cancer care. The company works closely with healthcare providers and researchers to deliver actionable genomic insights that help guide treatment decisions. Foundation Medicine’s goal is to accelerate the development of personalized therapies that target the specific genetic alterations driving cancer. With its focus on quality, scientific excellence, and regulatory leadership, Foundation Medicine is committed to improving the lives of cancer patients worldwide.
About FoundationOne®CDx:
FoundationOne®CDx is a comprehensive genomic profiling test that analyzes tumor DNA to identify genetic alterations. This test helps guide the selection of targeted therapies and provides tumor mutation profiling to inform clinical decisions. FoundationOne®CDx is intended for use by FDA healthcare professionals in oncology to identify patients who may benefit from specific treatments based on their tumor’s genetic profile.
