- Commercial-stage biotech Vanda Pharmaceuticals ( NASDAQ: VNDA ) announced Friday that the FDA issued Orphan Drug Designation for VPO-227, an experimental therapy for cholera.
- Despite the low incidence of cholera in the U.S., the infection is a global concern that leads to an estimated 1.4 – 4M cases and 21K – 143K deaths annually.
- VPO-227 is designed to block a cellular component called Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). CFTR can lead to cholera-induced intestinal fluid loss when it is affected by the cholera toxin.
- With its orphan drug designation, the FDA aims to offer financial incentives to drug developers targeting rare diseases and conditions.
- In addition to tax credits for clinical trial costs and waiver of the user fee for marketing applications, the developers can claim seven years of marketing exclusivity upon regulatory approval of orphan drugs.
For further details see:
Vanda granted FDA’s Orphan Drug Designation for cholera therapy