US FDA Awards RPD Status to Senhwa's Silmitasertib for Pediatric Neuroblastoma

TAIPEI and SAN DIEGO, Sept. 13, 2024 -- Senhwa Biosciences, Inc. (TPEx: 6492), a company specializing in developing novel therapeutics for oncology, rare, and infectious diseases, has announced that its drug Silmitasertib (CX-4945) has received a rare pediatric disease designation (RPDD) from the US FDA for treating neuroblastoma. This comes after Silmitasertib was previously granted RPDD for medulloblastoma in July 2020, underscoring its significant potential in combating rare pediatric cancers.

Silmitasertib is a pioneering small molecule drug that inhibits the CK2 protein, demonstrating antitumor properties in pre-clinical studies for neuroblastoma. The rare pediatric disease designation qualifies the drug for the priority review voucher (PRV) program, which aims to promote the development of new treatments for rare pediatric conditions. Silmitasertib has already received one RPDD and three orphan drug designations (ODD) from the FDA.

Jin-Ding Huang, PhD, CEO of Senhwa Biosciences, Inc., remarked, "The RPD designation provides sponsors with the added incentive of requesting priority review vouchers (PRVs) for future marketing applications. This program encourages the development of drugs for rare pediatric diseases."

Neuroblastoma is an embryonic tumor affecting the peripheral sympathetic nervous system and ranks as the third most common pediatric cancer and the leading cancer in infants. It is typically diagnosed in children around one to two years old, with approximately 700 to 800 new cases annually in the United States.

While the survival rates for low and intermediate-risk neuroblastoma are high, high-risk neuroblastoma has a five-year survival rate of about 50% and a high likelihood of recurrence, even with aggressive treatments. Currently, there is no established treatment for relapsed or refractory neuroblastoma.

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