Senhwa Biosciences, Inc. (TPEx: 6492), a new drug development company focusing on first-in-class therapeutics for oncology, rare diseases, and infectious diseases, today announced that its new drug Silmitasertib (CX-4945) was granted an orphan drug designation (ODD) by US FDA for the treatment of neuroblastoma. This follows the FDA’s acknowledge of Rare Pediatric Disease Designation (RPDD) to Silmitasertib (CX-4945) on September 12th.
“ We are pleased to receive orphan drug designation for Silmitasertib for the treatment of neuroblastoma, a rare, malignant pediatric disease for which there remain unmet medical needs. ODD represents an important regulatory milestone that can potentially expedite the clinical development of Silmitasertib, which is a potent and selective CK2 inhibitor,” said Jin-Ding Huang, PhD, CEO of Senhwa Biosciences Inc.
The US FDA grants ODD status to drugs and biologics that are intended to treat, prevent or diagnose a life-threatening or chronically debilitating rare disease with a prevalence of fewer than 200,000 people in the US. ODD affords certain financial incentives to support clinical development, including the potential for up to seven years of market exclusivity in the United States upon regulatory approval.
The granting of ODD indicates the significant potential of Silmitasertib (CX-4945) as a drug treating rare pediatric cancers. Additionally, Silmitasertib (CX-4945) has previously obtained orphan drug designation for several indications, including cholangiocarcinoma, medulloblastoma, and biliary tract cancer.
Neuroblastoma is the most common malignant solid tumor in children, aside from brain tumors and lymphomas, with over 90% diagnosed before the age of five. About 70% of affected children have already metastasized by the time symptoms appear, and the 20-year survival rate is only 30%. In the United States, there are an average of 700 to 800 new cases each year, accounting for approximately 6% of childhood cancers, meeting the definition of a rare disease.
The Phase I/II study of Silmitasertib (CX-4945) combined with chemo therapeutics in children and young adults with relapsed refractory solid tumors including neuroblastoma will be conducted by the Penn State Health Children’s Hospital and the prestigious Beat Childhood Cancer Research Consortium with Silmitasertib (CX-4945) drug supply supported by Senhwa Biosciences