First-in-Class, Oral Agent Gets Orphan Drug Status for Pancreatic Cancer

The Food and Drug Administration (FDA) has granted orphan drug designation to Mesupron (upamostat; RedHill Biopharma) for the adjuvant treatment of pancreatic cancer.

Mesupron is a first-in-class, orally-administered protease inhibitor; high levels of certain proteases have been associated with poor prognosis for various solid tumor cancers. The drug represents a non-cytotoxic approach to cancer therapy with several potential mechanism of action to inhibit tumor invasion and metastasis. 

Several Phase 1 and two Phase 2 proof of concept studies have already been conducted (one in patients with locally-advanced, unresectable pancreatic cancer, another in patients with metastatic breast cancer in combination with first-line chemotherapeutic agents). The Company also plans to evaluate Mesupron for the treatment of several inflammatory gastrointestinal disorders.

For more information visit www.RedHillBio.com.