CHOP Pioneers Helped Ensure Drug Is Available to Children

Physician-scientists at Children’s Hospital of Philadelphia (CHOP) are applauding accelerated approval by the U.S. Food and Drug Administration of a new drug to treat childhood cancer, Rozlytrek™ (entrectinib).

The drug targets a key genetic driver of cancer, rather than a specific type of tumor. It’s hailed as an important advance for children with cancers with genetic defects in the neurotrophic tyrosine receptor kinase (NTRK), including brain and solid tumors.

Garrett Brodeur, MD, and Elizabeth Fox, MD, were instrumental in assuring the drug approval included children.

Dr. Brodeur conducted pre-clinical studies specifically in childhood cancers. Based on this data, Dr. Fox and colleagues designed and launched the clinical trial, and expanded to a national and then global study treating children with solid tumors and brain tumors. Data from this clinical trial in children was one of the four trials used to support the FDA accelerated approval of Rozlytrek.

“After taking Rozlytrek, children with cancers, including brain tumors with NRTK fusions, experienced impressive decreases in their cancer burden with minimal side effects from this oral medication,” said Fox. “This accelerated FDA approval of Rozlytrek is a collaborative effort to make sure that the most promising new drugs are available not just to adults, but to children with cancer.”

Learn more about Rozlytrek.