FDA grants rare pediatric disease designation to treatment for medulloblastoma subtype

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease (RPD) designation for a Casein Kinase 2 (CK2) inhibitor (Silmitasertib, Senhwa Biosciences, Inc), for the treatment of recurrent sonic hedgehog (SHH) medulloblastoma. 

Recurrent SHH medulloblastoma is a major sub-group of medulloblastoma, a cancerous brain tumor in children. Less than 100 news cases of recurrent SHH medulloblastoma are diagnosed a year.

 A Phase 1/2 and Surgical study of Silmitasertib, in patients with recurrent SHH medulloblastoma is currently being conducted with the Pediatric Brain Tumor Consortium and funded through the Consortium grant awarded by the National Institute of Health.

Read the full press release here.