The FDA has granted Rare Pediatric Disease Designation and Orphan Drug Designation to Canget BioTekpharma LLC for FL118, an anticancer drug candidate discovered at Roswell Park Comprehensive Cancer Center, for the treatment of osteosarcoma. The small molecule, also known as 10,11-methylenedioxy-20(S)-camptothecin, is a camptothecin derivative based on a compound found in Xishu tree bark traditionally used in Chinese medicine. FL118 had previously received Orphan Drug Designation for pancreatic cancer, and the new pediatric and osteosarcoma-related designations broaden its regulatory framework for development in rare and difficult-to-treat cancers.
Osteosarcoma is a rare, aggressive bone cancer that primarily affects children, adolescents and young adults, with limited options for patients whose disease is recurrent, metastatic or resistant to current therapies. Orphan Drug Designation and Rare Pediatric Disease Designation are FDA programs intended to encourage development of treatments for rare and serious conditions, particularly those that largely affect children. If FL118 is ultimately approved for a qualifying rare pediatric indication and all statutory criteria are met, Canget could become eligible for a Priority Review Voucher.
FL118 was identified in the lab of Fengzhi Li, PhD, at Roswell Park, and preclinical studies suggest anticancer activity across multiple models as well as involvement in pathways linked to cancer survival and drug resistance. Researchers at Roswell Park note that FL118 may also provide a platform for related compounds targeting other rare or hard-to-treat tumors, including malignant pleural mesothelioma, and that ongoing mechanistic work is aimed at further characterizing how the agent exerts its effects.
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