Vanda Pharmaceuticals Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for their novel therapeutic candidate, VGT-1849B, intended for the treatment of polycythemia vera (PV). This designation aims to facilitate the development of this selective peptide nucleic acid-based JAK2 inhibitor, which targets the JAK2 V617F mutation prevalent in over 95% of PV patients. The Orphan Drug Designation provides benefits to drug developers working on treatments for rare medical conditions. VGT-1849B is designed to offer targeted efficacy with an improved safety profile and convenient infrequent dosing, potentially enhancing the quality of life for patients with PV.
Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Vanda Pharmaceuticals Inc. published the original content used to generate this news brief via PR Newswire (Ref. ID: PH60962) on August 28, 2025, and is solely responsible for the information contained therein.