Clinical stage drug development company focused on next generation DNA Damage Response (DDR) therapeutics Senhwa Biosciences, Inc. (TPEx: 6492) announced on Friday that its drug Silmitasertib (CX-4945) received Rare Paediatric Disease Designation (RPDD) from the US FDA for neuroblastoma treatment.

This follows a previous RPDD granted for medulloblastoma in July 2020, underscoring Silmitasertib's potential in rare pediatric cancers. Silmitasertib, a first-in-class CK2 inhibitor, demonstrated antitumor activity in pre-clinical neuroblastoma studies.

The RPDD qualifies the drug for the Priority Review Voucher (PRV) program, designed to accelerate the development of novel therapies for rare pediatric diseases. Silmitasertib has already been granted one RPDD and three Orphan Drug Designations (ODD) by the FDA.

Neuroblastoma, predominantly affecting infants and diagnosed on average between 1 and 2 years old, represents the third most common pediatric cancer in the US, with 700 to 800 new cases annually. High-risk neuroblastoma has a 5-year survival rate of approximately 50% and is prone to recurrence despite intensive treatment; no standard treatment exists for relapsed or refractory cases.