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VGT-1849B granted orphan drug designation by the FDA for PV
On August 28, 2025, the U.S. Food and Drug Administration granted orphan drug designation to VGT-1849B, a selective peptide nucleic acid-based Janus kinase (JAK)2 inhibitor, for the treatment of patients with polycythemia vera.1
VGT-1849B is an antisense oligonucleotide with a modified peptide nucleic acid backbone, designed for enhanced cell permeability and ribonucleic acid affinity.1 While several JAK inhibitors are approved for the treatment of patients with polycythemia vera, VGT-1849B is the first agent to solely target JAK2, aiming to reduce the risk of infections and other adverse effects associated with inhibition of other JAKs and non-JAKs.1
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What would be your preferred strategy to treat patients with newly diagnosed MF with moderate anemia (Hb <10 g/dL) and platelet count <200,000/µL?