Vanda Pharmaceuticals Announces FDA Granted Orphan Drug Designation for VGT-1849B, a Novel and Selective Candidate for the Treatment of Polycythemia Vera

Core Viewpoint - Vanda Pharmaceuticals Inc. has received Orphan Drug Designation from the FDA for VGT-1849B, a selective JAK2 inhibitor aimed at treating polycythemia vera (PV) [1][5]. Group 1: Product Overview - VGT-1849B is a selective peptide nucleic acid-based JAK2 inhibitor designed to target JAK2 mRNA, thereby reducing JAK2 protein production and downstream signaling associated with PV [3][8]. - The prevalence of PV in the U.S. is estimated to affect 44 to 57 per 100,000 people, with over 95% of patients harboring the JAK2 V617F mutation [2][9]. - VGT-1849B utilizes a novel backbone chemistry, OliPass Peptide Nucleic Acid (OPNA), which enhances cell permeability and RNA affinity [2][8]. Group 2: Mechanism of Action - By selectively targeting JAK2 mRNA, VGT-1849B effectively reduces JAK2-driven cell proliferation and suppresses hematopoiesis, leading to decreased production of red blood cells, neutrophils, platelets, and lymphocytes [3][4]. - The drug aims to provide a favorable safety profile by avoiding off-target effects commonly associated with other JAK inhibitors [4]. Group 3: Market Context - Current JAK2 inhibitors on the market, such as Jakafi®, Inrebic®, Ojjaara®, and Vonjo®, are not solely selective to JAK2, which can lead to increased toxicity [4]. - If approved, VGT-1849B could offer targeted efficacy with an improved safety profile and convenient infrequent dosing, addressing a significant unmet medical need in the treatment of PV [5]. Group 4: Company Background - Vanda Pharmaceuticals Inc. is focused on developing innovative therapies to meet high unmet medical needs and improve patient lives [7].