Vanda Pharmaceuticals Inc. announced on August 28, 2025, that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VGT-1849B. This novel candidate is a selective peptide nucleic acid-based JAK2 inhibitor for the treatment of polycythemia vera (PV).
Polycythemia vera is a chronic myeloproliferative disorder affecting an estimated 44 to 57 per 100,000 people in the United States. Over 95% of PV patients harbor the JAK2 V617F gain-of-function mutation, leading to aberrant JAK2 production. VGT-1849B utilizes OliPass Peptide Nucleic Acid (OPNA) chemistry, a novel backbone designed to enhance cell permeability and RNA affinity.
By selectively targeting JAK2 mRNA, VGT-1849B aims to reduce JAK2 protein production and V617F-driven autonomous cell proliferation, without off-target kinase effects. This approach is intended to offer targeted efficacy with an improved safety profile and convenient infrequent dosing compared to existing JAK inhibitors, addressing the disease burden for PV patients.
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