Napabucasin has been granted Orphan Drug Designation from the FDA in the treatment of gastric cancer, including gastroesophageal junction (GEJ) cancer. Napabucasin is an orally administered cancer stemness inhibitor designed to inhibit cancer stemness pathways by targeting STAT3, and is currently being investigated in a global Phase III clinical trial in combination with paclitaxel in patients with advanced gastric and GEJ cancer (the BRIGHTER study).
The FDA's Orphan Drug Designation program provides special status and development incentives for drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Each year, about 26,000 people in the U.S. are diagnosed with gastric cancer.
Phase Ib/II data for napabucasin in gastric/GEJ cancer were previously presented at the American Society of Clinical Oncology 2015 Annual Meeting; these data found that napabucasin and weekly paclitaxel can be combined in patients with advanced pre-treated gastric/GEJ cancer. Lesion regression, objective responses and prolonged stable disease were observed in heavily pre-treated patients. Common adverse events identified through this clinical trial included grade 1 or 2 diarrhea, nausea, fatigue, abdominal pain, vomiting and anorexia