Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) announced on Friday that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for VPO-227 for the treatment of cholera, a widespread infectious diarrheal disease with estimated 1.4 to 4 million cases worldwide, resulting in 21,000 to 143,000 deaths annually.

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.

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.