Orphan Status for Biliary Tract Cancer Drug

FDA has granted Nuvectis Pharma’s NXP800 an orphan drug designation for treating cholangiocarcinoma. The company describes NXP800 as an oral small molecule with a novel mechanism of action that has “demonstrated robust activity in several preclinical cancer models, including ARID1a-mutated ovarian, endometrial and gastric carcinomas, as well as cholangiocarcinoma.”

Cholangiocarcinoma is a biliary tract cancer originating in the epithelium of the biliary tree. “Surgical resection is the only potentially curative treatment for cholangiocarcinoma, but the disease is often diagnosed as unresectable because of local extension and/or metastases,” the company says.

NXP800 is currently being evaluated in a Phase 1b clinical trial in patients with platinum resistant, ARID1a-mutated ovarian carcinoma, with additional diseases planned for future clinical trials, Nuvectis says.