The US Food and Drug Administration (FDA) has granted orphan drug designation for VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor for the treatment of polycythemia vera.
VGT-1849A is a novel ASO treatment candidate for polycythemia vera and other JAK2-driven hematologic malignancies that, if approved, could offer targeted efficacy with an improved safety profile and convenient dosing.
More than 95% of patients with polycythemia vera harbor the JAK2 V617F gain-of-function mutation leading to aberrant JAK2 production. By selective reduction of JAK2 levels, VGT-1849A has the potential to reduce JAK2V617F-driven pathogenic signaling, ultimately suppressing the malignant proliferation and survival of hematopoietic cells, according to the drug’s manufacturer Vanda Pharmaceuticals.
By specifically targeting JAK2, VGT-1849A seeks to reduce the risk of infection and toxic effects associated with the use of currently available small molecule inhibitors that lack sole selectivity for the target JAK2 protein kinase, the company reported in a press release.
ASOs such as VGT-1849A have broad applicability in addressing a number of disorders caused by genetic variants, Vanda wrote.
