The FDA granted orphan drug designation to silmitasertib for treatment of medulloblastoma.
Silmitasertib (CX-4945, Senhwa Biosciences) is a highly selective inhibitor of casein kinase 2 (CK2).
The Pediatric Brain Tumor Consortium is conducting a phase 1/phase 2 and surgical study of silmitasertib for both adults and children with recurrent sonic hedgehog-driven medulloblastoma.
“We are pleased to receive orphan drug designation for silmitasertib for medulloblastoma, a rare, severe pediatric disease for which there are no approved targeted therapies,” John Soong, MD, FCAP, chief medical officer of Senhwa Biosciences, said in a company-issued press release. “Orphan drug designation represents an important regulatory milestone that has the potential to expedite the clinical development of silmitasertib.”
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
The FDA previously granted the agent fast track and rare pediatric disease designations for medulloblastoma indications.