The FDA has granted the Orphan Drug Designation to VGT-1849A, a novel Janus kinase 2 (JAK2) inhibitor for the treatment of patients with polycythemia vera (PV), according to a press release from Vanda Pharmaceuticals.
“If approved, VGT-1849A could offer targeted efficacy with an improved safety profile and convenient dosing,” Vanda writes.
VGT-1849A is an antisense oligonucleotide-based JAK2 inhibitor that is designed to have less off-target suppression of JAK1, JAK3, TYK2, and other kinases than currently available small molecule JAK2 inhibitors.
“This orphan designation for VGT-1849A is an important milestone in precision medicine-based therapeutics in the space of hematological malignancies,” said Mihael Polymeropoulos, MD, President, CEO, and chairman of the board at Vanda. “This milestone marks the second precision medicine therapeutic for Vanda following the development of VCA-894A for Charcot-Marie-Tooth that is expected to begin clinical testing in the coming months.”
The FDA Orphan Drug Designation is granted to novel drugs or biologic products that show potential to prevent, diagnose, or treat rare diseases or conditions. VCA-894A was granted the Orphan Drug Designation in June 2023.
Vanda Pharmaceuticals announces orphan drug designation granted for VGT-1849A, a novel and selective antisense oligonucleotide candidate for the treatment of polycythemia vera. News release. December 20, 2024. Accessed January 6, 2025. https://bit.ly/40a1f3A
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