The US Food and Drug Administration (FDA) has granted orphan drug status to Radiopharm Theranostics Ltd (ASX:RAD)’s Ga68-Trivehexin (RAD 301) radiopharmaceutical technology for imaging of patients with pancreatic ductal adenocarcinoma (PDAC).
Radiopharm is developing Trivehexin as a novel radiopharmaceutical for imaging and treatment of pancreatic cancer, with potential follow-on applications for indications including non-small cell lung cancer, head and neck and colorectal cancers.
High unmet need
Pancreatic ductal adenocarcinoma is the primary targeted indication because of it presents a high unmet need.
Trivehexin is a proprietary peptide-based molecule that targets αvβ6-integrin, a cellular marker for tumour invasion and metastatic growth, the expression of which correlates with decreased survival in several carcinomas.
The αvβ6-integrin receptor is found in high density on most pancreatic carcinoma cells, making it an attractive diagnostic and therapeutic target.
“Orphan Drug Designation for RAD 301 comes on top of FDA IND approval for a Phase 1 clinical trial in pancreatic cancer, which is planned to start in the next few weeks in the United States,” said CEO and managing director Riccardo Canevari.
“This important designation further reinforces the excitement of investigators conducting the study.
“The FDA’s decision highlights the significant demand for effective imaging agents for improved and earlier diagnosis of pancreatic cancer, which has one of the highest levels of unmet needs among all cancer types.”
Two orphan drug designations
As a result of the decision the company now holds two FDA orphan drug designations, along with the LRRC15 antibody DUNP19 for the treatment of patients with osteosarcoma.
Designation is granted for a drug or biologic product with the potential to diagnose, prevent or treat rare diseases and conditions.
Recipients of the designation receive benefits and incentives including tax credits for qualified clinical trials, exemption from user fees and a potential seven years of market exclusivity if the drug is approved.
Radiopharm has signed an exclusive licensing agreement with TRIMT GmbH for the development and commercialisation of RAD 301 in USA, Australia, China, Hong Kong and Japan.