AbbVie Gets FDA Rare Pediatric Disease Designation for Investigational ABT-414

AbbVie Inc.

revealed Monday that the Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation for its ABT-414, an investigational antibody drug conjugate (ADC) focused on the epidermal growth factor receptor (EGFR). The drug candidate is for the treatment of pediatric patients with EGFR-amplified Diffuse Intrinsic Pontine Glioma (DIPG). That was known to be highly aggressive, as well as, difficult to treat brain tumors found at the base of the brain. AbbVie said that though brainstem tumors were extremely rare among adults, the company indicated that they consisted of 10-15 percent of all pediatric brain tumors. The company added that DIPG was 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 United States. The company pointed out that the medical regulator granted the Rare Pediatric Disease Designation based on a planned pediatric sub-study "nested" within the ongoing Phase 2 trial of ABT-414 in adults with recurrent EGFR-amplified glioblastoma. That was conducted in alliance with the European Organization for Research and Treatment of Cancer (EORTC). AbbVie VP for Oncology clinical development, Gary Gordon, said, "Pediatric patients with high grade gliomas have a rare and fatal disease. This Rare Pediatric Designation, a first for AbbVie, is an important advancement as we continue to evaluate ABT-414 and its potential to help this group of patients who desperately need a new treatment option. The proposal of including a nested cohort within an adult global trial is an endeavor that we hope may bring more treatments to pediatric patients." The stock advanced 0.14 percent in the pre-market trading on Monday.