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The FDA granted Rare Pediatric Disease Designation for ABT-414, an investigational antibody drug conjugate targeting the epidermal growth factor receptor (EGFR) for the treatment of pediatric patients with EGFR-amplified Diffuse Intrinsic Pontine Glioma (DIPG), known to be highly aggressive and difficult to treat brain tumors found at the base of the brain.

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Though brainstem tumors are extremely rare among adults, they comprise approximately 10-15 percent of all pediatric brain tumors. DIPG is the most common subtype of tumor in this anatomical region and the second most common malignant brain tumor of childhood, with an estimated 200-400 children affected each year in the U.S.


The FDA granted the Rare Pediatric Disease Designation based on a proposed pediatric sub-study "nested" within the ongoing phase II study of ABT-414 in adults with recurrent EGFR-amplified glioblastoma, conducted in collaboration with the European Organization for Research and Treatment of Cancer.