Vanda Pharmaceuticals Announces Orphan Drug Designation Granted for VPO-227, a Novel Candidate for the Treatment of Cholera
CollaborateOrphan DrugSmall molecular drugPriority Review
WASHINGTON, Oct. 21, 2022 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VPO-227 for the treatment of cholera.
While the incidence of cholera in the U.S. is low, cholera is a widespread infectious diarrheal disease with estimated 1.4 to 4 million cases worldwide, resulting in 21,000 to 143,000 deaths annually.1 Many of these deaths occur in children, as diarrheal diseases are the second leading cause of death globally among children under 5 years of age.2 Cholera remains a major public health challenge and recognized unmet medical need despite decades-long public health efforts.
"This designation is an important milestone in the development of VPO-227 and highlights the need for potential new treatment options for patients who suffer from cholera and other conditions of water hyper-excretion," said Mihael H. Polymeropoulos, M.D., Vanda's President, CEO and Chairman of the Board.
VPO-227 is a small-molecule with a novel mechanism of action which blocks the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) ion channel. CFTR plays a pivotal role in cholera-induced intestinal fluid loss as it is affected by the cholera toxin. VPO-227, therefore, has the potential to be an orally administered treatment for cholera. In a recent publication in the journal Toxins, the authors showed that oral VPO-227 protects animals from a lethal cholera challenge using two separate cholera strains.3
Orphan Drug Designation is granted by the FDA to investigational therapies addressing rare medical conditions and provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and the potential for seven years of post-approval marketing exclusivity.
Additionally, in order to incentivize development in tropical diseases, including cholera, the FDA has established the Tropical Disease Priority Review Voucher Program, which grants a transferable Priority Review Voucher to sponsors of eligible new drug applications for products treating tropical diseases. Vanda expects to submit an Investigational New Drug (IND) application to the FDA for VPO-227 in 2023.
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. For more on Vanda Pharmaceuticals Inc., please visit www.vandapharma.com and follow us on Twitter @vandapharma.
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.
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