AbbVie, a global biopharmaceutical company, announced that the FDA granted Rare Pediatric Disease Designation for ABT-414, an investigational antibody drug conjugate (ADC) 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.

Though brainstem tumors are extremely rare among adults, they comprise approximately 10-15% 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 (EORTC).

"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," said Gary Gordon, M.D., vice president, oncology clinical development, AbbVie. "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."

ABT-414 is an investigational monoclonal antibody drug conjugate (ADC) targeting the epidermal growth factor receptor (EGFR) developed by AbbVie researchers with components in-licensed from Life Science Pharmaceuticals and Seattle Genetics. AbbVie is evaluating it for the treatment of adult patients with EGFR-amplified glioblastoma, an aggressive malignant primary brain tumor. In 2014, the FDA and the EMA granted Orphan Drug Designation for the treatment of adult glioblastoma and glioma, respectively. In 2016, the FDA granted Rare Disease Designation to ABT-414 for the treatment of pediatric patients with EGFR-amplified Diffuse Intrinsic Pontine Gliomas (DIPG). ABT-414 is an investigational compound and its efficacy and safety have not been established by the FDA or any other health authority.

Diffuse Intrinsic Pontine Glioma (DIPG) is a type of high grade glioma (HGG). HGG represents one of the most common central nervous system (CNS) tumors among adults; however, they comprise only approximately 8-12% of all CNS tumors in pediatric patients. Pediatric HGG are highly aggressive tumors that account for a significant amount of morbidity and mortality among children. Despite numerous treatment approaches, five-year survival outcomes range from 15-35% and the majority of children succumb to their disease.

Glioblastoma is the most common and most aggressive type of malignant primary brain tumor. Prior to diagnosis, patients may experience headache, vision problems, nausea/vomiting, personality changes and seizures. For adults with more aggressive glioblastoma, treated with concurrent Temozolamide and radiation therapy, median survival is about 14.6 months. Treatment for glioblastoma remains challenging. Standard treatment is surgical resection, radiotherapy and concomitant adjunctive chemotherapy.