FL118 Granted FDA Rare Pediatric Disease Designation and Orphan Drug Designation for Osteosarcoma

BUFFALO, N.Y. — The U.S. Food and Drug Administration has granted Rare Pediatric Disease Designation and Orphan Drug Designation to Canget BioTekpharma LLC, a Roswell Park spinoff company, for FL118, a novel anticancer drug candidate discovered at Roswell Park Comprehensive Cancer Center, for the treatment of osteosarcoma.

Together, the designations strengthen the development position of FL118 and may help support future clinical, translational and strategic collaboration efforts aimed at advancing new treatment options for patients with osteosarcoma and other rare cancers.

FL118, also known by its chemical name 10,11-methylenedioxy-20(S)-camptothecin (10,11-MD-CPT), is a novel camptothecin-derived small molecule based on a compound found in Xishu tree bark, and used in traditional Chinese medicine. FL118 was previously granted FDA Orphan Drug Designation for the treatment of pancreatic cancer, and the new osteosarcoma-related designations further expand the regulatory and development foundation for FL118 in rare and difficult-to-treat cancers. 

Osteosarcoma is a rare, aggressive bone cancer that most commonly affects children, adolescents and young adults. Despite advances in surgery and chemotherapy, treatment options remain limited for patients with recurrent, metastatic or drug-resistant disease, highlighting the need for new therapeutic approaches.

Orphan status is a designation awarded by the FDA to encourage research and development of drugs that have shown promise in treating, preventing or diagnosing rare diseases. The FDA’s Rare Pediatric Disease Designation program is designed to encourage development of treatments for serious or life-threatening diseases that primarily affect children. If FL118 is ultimately approved for a qualifying rare pediatric disease indication and all statutory requirements are met, Canget could be eligible to receive a Priority Review Voucher (PRV).

“Receiving both Rare Pediatric Disease Designation and Orphan Drug Designation for FL118 in osteosarcoma is an important milestone for this Roswell Park-discovered compound,” says Fengzhi Li, PhD, Associate Professor of Oncology in the Department of Pharmacology and Therapeutics at Roswell Park and founder of Canget BioTekpharma. “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.”

Discovered by a team of scientists led by Dr. Li, FL118 has shown broad and significant anticancer activity in preclinical studies and may play a role in key cancer-survival and drug-resistance pathways. Growing evidence also suggests that FL118 may provide a novel drug platform for developing related compounds against additional rare or difficult-to-treat cancers 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,” adds Xiang Ling, MD, PhD, a senior researcher on the FL118 team at Roswell Park. 

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From the world’s first chemotherapy research to the PSA prostate cancer biomarker, Roswell Park Comprehensive Cancer Center generates innovations that shape how cancer is detected, treated and prevented worldwide. Driven to eliminate cancer’s grip on humanity, the Roswell Park team of 4,000 makes compassionate, patient-centered cancer care and services accessible across New York State and beyond. Founded in 1898, Roswell Park was among the first three cancer centers nationwide to become a National Cancer Institute-designated comprehensive cancer center and is the only one to hold this designation in Upstate New York. To learn more about Roswell Park Comprehensive Cancer Center and the Roswell Park Care Network, visit www.roswellpark.org, call 1-800-ROSWELL (1-800-767-9355) or email ASKRoswell@RoswellPark.org.