Senhwa Biosciences announced today that its investigational drug, Silmitasertib (CX-4945), has received Orphan Drug Designation (ODD) from the U.S. FDA for a new indication in treating neuroblastoma. This follows the recent recognition of Silmitasertib under the FDA's Rare Pediatric Disease Designation (RPDD). Currently, Silmitasertib has ODD status for multiple indications, including cholangiocarcinoma, medulloblastoma, and biliary tract cancer. With this designation, the FDA offers administrative support, market exclusivity, and other incentives to facilitate development. Upon approval, the drug would benefit from a seven-year period of exclusive marketing rights in the U.S. market.

The FDA’s Orphan Drug Designation stems from the Orphan Drug Act (ODA), which was established to encourage the development of treatments for rare diseases affecting fewer than 200,000 patients in the U.S. Drugs with ODD status benefit from tax credits for clinical testing expenses, reduced Prescription Drug User Fees, and potential market exclusivity of seven years. Market exclusivity is granted if the drug is the first approved treatment for that indication or demonstrates superior efficacy over existing therapies.

According to Senhwa Biosciences, the FDA’s recognition of Silmitasertib (CX-4945) as an orphan drug for various rare pediatric diseases and cancers highlights its significant potential in addressing unmet medical needs. Neuroblastoma is among the most common malignant solid tumors in children, second only to brain tumors and lymphomas. Over 90% of cases are diagnosed before the age of five, with around 70% of children presenting with metastasis at diagnosis and a survival rate of just 30% over 20 years. In the U.S., approximately 700 to 800 new cases of neuroblastoma are diagnosed annually, accounting for about 6% of pediatric cancers and meeting the criteria for a rare disease.

Senhwa Biosciences has partnered with Penn State Health Children’s Hospital and the renowned Beat Childhood Cancer Research Consortium for a Phase I/II clinical trial to evaluate Silmitasertib (CX-4945) in combination with chemotherapy for recurrent or refractory solid tumors in children and adolescents. The trial, which has received FDA approval, is progressing smoothly.

Resource (mandarin): 生華科CX-4945再獲ODD資格，新適應症神經母細胞瘤上市可獲7年獨賣期