FDA News

FDA grants rare pediatric disease designation to ABT-414 for DIPG

The FDA granted rare pediatric disease designation to ABT-414 for the treatment of children with epidermal growth factor receptor–amplified diffuse intrinsic pontine glioma.

Diffuse intrinsic pontine glioma (DIPG) — a highly aggressive and difficult-to-treat brain tumor located at the base of the brain — is the second most common malignant pediatric brain tumor, affecting approximately 200 to 400 children each year in the United States.

The designation was based on a proposed pediatric substudy “nested” within an ongoing phase 2 study of ABT-414 (AbbVie, Life Science Pharmaceuticals, Seattle Genetics) — an investigational antibody drug conjugate targeting EGFR — in adults with recurrent EGFR–amplified glioblastoma.

“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,” Gary Gordon, MD, vice president of oncology clinical development for AbbVie, said in a press release. “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 received orphan drug designation from the FDA for the treatment of adults with glioblastoma in 2014.

The FDA granted rare pediatric disease designation to ABT-414 for the treatment of children with epidermal growth factor receptor–amplified diffuse intrinsic pontine glioma.

Diffuse intrinsic pontine glioma (DIPG) — a highly aggressive and difficult-to-treat brain tumor located at the base of the brain — is the second most common malignant pediatric brain tumor, affecting approximately 200 to 400 children each year in the United States.

The designation was based on a proposed pediatric substudy “nested” within an ongoing phase 2 study of ABT-414 (AbbVie, Life Science Pharmaceuticals, Seattle Genetics) — an investigational antibody drug conjugate targeting EGFR — in adults with recurrent EGFR–amplified glioblastoma.

“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,” Gary Gordon, MD, vice president of oncology clinical development for AbbVie, said in a press release. “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 received orphan drug designation from the FDA for the treatment of adults with glioblastoma in 2014.