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

Core Insights - Vanda Pharmaceuticals Inc. has received Orphan Drug Designation from the FDA for VGT1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor aimed at treating polycythemia vera (PV), a rare hematologic malignancy affecting approximately 1 in 2000 Americans [1][8] - The ASO VGT1849A selectively reduces JAK2 levels, potentially suppressing JAK2V617F-driven pathogenic signaling and malignant proliferation of hematopoietic cells [1][9] - This designation represents a significant milestone in precision medicine for hematological malignancies, marking Vanda's second precision medicine therapeutic following VCA-894A for Charcot-Marie-Tooth disease [2] Company Overview - Vanda Pharmaceuticals is focused on developing innovative therapies to meet high unmet medical needs and improve patient lives [16] - The company aims to provide targeted efficacy with an improved safety profile through its selective targeting of JAK2, differentiating itself from currently available small molecule inhibitors that may have off-target effects [15] Product Details - VGT1849A is designed to alleviate the disease burden faced by PV patients by selectively targeting JAK2, which is implicated in over 95% of PV cases due to the JAK2 V617F mutation [8][9] - The potential benefits of VGT1849A include a favorable safety profile and convenient dosing, which may enhance the quality of life for patients with PV [8][9]