Senhwa Biosciences announced today (13th) that its new drug under development, Silmitasertib (CX-4945), has once again been granted the Rare Pediatric Disease Designation (RPDD) by the U.S. FDA, this time for the treatment of neuroblastoma. This designation facilitates the drug’s entry into the priority review pathway and potentially enhances its future market price upon approval.
The U.S. FDA encourages pharmaceutical companies to develop drugs for rare pediatric diseases. When a new drug receives the RPDD, it qualifies for a Priority Review Voucher (PRV), which shortens the approval review period to just six months. Additionally, the PRV is a tradable asset, allowing companies to sell it to other pharmaceutical firms. Publicly available information shows that Day One Biopharmaceuticals sold its PRV for an impressive $1.08 billion to an undisclosed buyer in May of this year. Similarly, French vaccine company Valneva sold its PRV for $1.03 billion in February.
According to Senhwa, rare pediatric diseases in the U.S. are defined as conditions affecting fewer than 200,000 patients nationwide, with over 50% of the affected population being infants, children, and adolescents aged 0-18. Neuroblastoma is the most common tumor among children, second only to brain tumors and lymphomas in incidence. The U.S. records only 700-800 new cases of neuroblastoma each year, making it a rare pediatric disease.
Silmitasertib (CX-4945) is a human protein kinase CK2 inhibitor with unique therapeutic advantages. By targeting and inhibiting CK2, a crucial protein, the drug effectively curbs the proliferation and survival of tumor cells. Preclinical studies have demonstrated Silmitasertib's significant efficacy in treating various hard-to-treat pediatric tumors. Prior to receiving the RPDD for neuroblastoma, Silmitasertib (CX-4945) had already been granted the Rare Pediatric Disease Designation for pediatric brain cancer—medulloblastoma—and orphan drug designation for three indications. These designations will help expedite the drug’s approval process, expand its indications, and secure a seven-year market exclusivity period in the U.S. after its approval.
Resource (mandarin): 生華科新藥再獲兒科罕病認定，助力加速上市審查資格
