May 29, 2024
Pancreatic Cancer

New Drug Candidate Receives FDA Orphan Drug Status for Pancreatic Cancer

Canget BioTekpharma LLC has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for their drug candidate, FL118, as a potential treatment for pancreatic cancer. FL118, also known as 10,11-methylenedioxy-20(S)-camptothecin (10,11-MD-CPT), has been developed at the Roswell Park Comprehensive Cancer Center. This designation is a significant step towards advancing the development and evaluation of new treatments for rare diseases.

Pancreatic cancer is considered a rare disease, affecting a relatively small number of people. This often results in limited treatment options compared to more common diseases. With only 3% of all cancers in the U.S. being pancreatic cancer, it is projected to become the second leading cause of cancer-related deaths in the country by 2030.

FL118 was discovered by a team of scientists at Roswell Park, led by Dr. Fengzhi Li, Associate Professor of Oncology in the Department of Pharmacology and Therapeutics. In preclinical studies, FL118 has demonstrated its ability to eliminate both pancreatic and colorectal tumor cells by targeting DDX5, a cancer-causing protein. Derived from camptothecin, a natural compound found in the bark and stem of a Chinese tree, FL118 has been used as a traditional Chinese remedy for centuries.

The Orphan Drug Designation granted by the FDA provides various incentives to encourage the development and evaluation of treatments for rare diseases. These incentives include tax credits for clinical trials, exclusive marketing rights, and exemptions from user fees. Currently, there have been only 206 pharmaceutical agents granted Orphan Drug Designation for pancreatic cancer since the FDA initiated the program in 1984.

By granting Canget BioTekpharma LLC this status, the FDA aims to speed up and support research efforts in finding effective treatments for pancreatic cancer. The designation serves as recognition of the potential impact FL118 may have in addressing the limited treatment options currently available for this disease.

Canget BioTekpharma LLC is now focused on further developing FL118 and conducting clinical trials to evaluate its safety and efficacy. The recent publication by Dr. Li and colleagues in the Journal of Medicinal Chemistry highlights the synthesis, identification, and characterization of new analogs of FL118, which have shown enhanced antitumor activity. These findings provide an exciting avenue for future research and potential advancements in pancreatic cancer treatment.

Overall, the granting of Orphan Drug Designation for FL118 is a significant milestone in the development of a novel treatment for pancreatic cancer. With limited treatment options currently available, the advancements made by Canget BioTekpharma LLC and Roswell Park Comprehensive Cancer Center pave the way for new hope and improved outcomes for patients with this rare and devastating disease.

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1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it