Vanda Pharmaceuticals Gets FDA Orphan Designation for VGT-1849B

 Vanda Pharmaceuticals has won a key U.S. Food and Drug Administration designation for its proposed VGT-1849B treatment of the rare blood disease polycythemia vera.

Vanda on Thursday said the FDA granted orphan-drug designation to VGT-1849B, which the Washington, D.C., biopharmaceutical company said selectively targets JAK2 and reduces JAK2 protein production without any off-target kinase effects.

The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S. It also provides for an extended marketing exclusivity period against competition.

Vanda said that while there are several JAK inhibitors available, none are solely selective to JAK2, adding that VGT-1849B is designed to reduce the risk of infection and toxic effects that are seen with inhibitors that also block JAK1, JAK3, TYK2 or other kinases outside of the JAK family.

https://www.morningstar.com/news/dow-jones/202508284863/vanda-pharmaceuticals-gets-fda-orphan-designation-for-vgt-1849-billion