Osteosarcoma Drug Earns Rare Pediatric Disease, Orphan Drug Designations
The FDA has granted rare pediatric disease designation and orphan drug designation to FL118 for the treatment of patients with osteosarcoma, according to a press release from Roswell Park.FL118, otherwise known by its chemical name 10,11-methylenedioxy-20(S)-camptothecin, is a small molecule inhibitor that represents a novel approach to addressing therapeutic resistance in rare and aggressive malignancies.Specifically, it is a camptothecin derivative based on a compound traditionally found in Xishu tree bark.
Preclinical Findings and Broad Potential
Reportedly, the regulatory decisions were supported by preclinical findings demonstrating that FL118 exhibits antitumor activity across multiple cancer models, with the potential to play a role in both cancer-survival and drug-resistance pathways.Furthermore, preclinical assessments have indicated that the agent provides a platform for treating other difficult-to-manage rare tumors, such as malignant pleural mesothelioma.
“FL118 has demonstrated unexpected anticancer activity with a favorable preclinical toxicity profile, and our mechanistic studies continue to reveal important insights into how this compound works,” said Xiang Ling, MD, PhD, a senior researcher on the FL118 team in the Department of Pharmacology & Therapeutics at Roswell Park, in the press release.
Regulatory Incentives
Under the provisions of the rare pediatric disease designation, if a new drug application for FL118 is accepted for the treatment of osteosarcoma, the developer may become eligible to receive a priority review voucher from the FDA.
“Receiving both rare pediatric disease designation and orphan drug designation for FL118 in osteosarcoma is an important milestone for this Roswell Park-discovered compound,” stated Fengzhi Li, PhD, associate professor of oncology in the Department of Pharmacology and Therapeutics at Roswell Park and founder of Canget BioTekpharma, in the press release.“These designations recognize the unmet medical needs in osteosarcoma and provide meaningful regulatory incentives that may help advance FL118 toward further development for pediatric and rare cancers.”
