By Chris Wack
Vanda Pharmaceuticals said the Food and Drug Administration has granted orphan-drug designation for VGT-1849A.
VGT-1849A is a selective antisense oligonucleotide-based JAK2 inhibitor for the treatment of polycythemia vera, a form of a rare hematologic malignancy.
The biopharmaceutical company said ASO VGT-1849A has the potential to reduce JAK2V617F-driven pathogenic signaling, ultimately suppressing the malignant proliferation and survival of hematopoietic cells.
By specifically targeting JAK2, the Washington, D.C., company hopes to reduce the risk of infection and toxic effects that are seen with inhibitors also blocking JAK1, JAK3, TYK2, or other kinases outside of the JAK family.
If approved, Vanda said VGT-1849A could offer targeted efficacy with an improved safety profile and convenient dosing.
Orphan-drug designation is granted by the FDA to investigational therapies addressing rare medical conditions and provides benefits to drug developers.
Write to Chris Wack at chris.wack@wsj.com
(END) Dow Jones Newswires
December 20, 2024 09:37 ET (14:37 GMT)
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