Vanda Pharmaceuticals Gains Orphan Drug Status for Investigational Cholera Drug
Vanda Pharmaceuticals’ VPO-227 has received FDA’s Orphan Drug designation for the treatment of patients with cholera.
VPO-227 is a small molecule with a novel mechanism of action, which blocks the cystic fibrosis transmembrane conductance regulator (CFTR) ion channel, which plays a role in cholera-induced intestinal fluid loss.
The company plans on submitting an Investigational New Drug application for the drug candidate in 2023.
The Orphan Drug designation exempts drug sponsors from FDA user fees and potentially offers them seven years of market exclusivity after a drug’s approval.
About VPO-227
VPO-227 (formerly BPO-27) is a small molecule CFTR inhibitor in development for the treatment of secretory disorders, including cholera. Vanda entered into a license agreement with University of California, San Francisco (UCSF) in 2017, under which Vanda acquired an exclusive worldwide license from UCSF to develop and commercialize a portfolio of CFTR activators and inhibitors, including the activator VPO-227. CFTR activators and inhibitors may have broad applicability in addressing a number of disorders, including chronic dry eye, constipation, polycystic kidney disease, cholestasis, and secretory diarrheas.
Vanda Pharmaceuticals Inc.
Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients. Headquarters: Washington, D.C., United States. Founded: 2002. Subsidiaries: Vanda Pharmaceuticals Netherlands B.V., Vanda Pharmaceuticals Limited.
October 26, 2022
