VGT-1849A, an antisense oligonucleotide-based novel Janus kinase 2 (JAK2) inhibitor, has received the Orphan Drug Designation from the FDA for management of polycythemia vera (PV). This was announced in a press release from Vanda Pharmaceuticals.
“If approved, VGT-1849A could offer targeted efficacy with an improved safety profile and convenient dosing,” Vanda writes.
As compared with currently available small molecule JAK2 inhibitors, VGT-1849A features reduced off-target suppression of JAK1, JAK3, TYK2, and other kinases.
“This orphan designation for VGT-1849A is an important milestone in precision medicine-based therapeutics in the space of hematological malignancies,” remarked 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 grants the Orphan Drug Designation to novel drugs or biologic agents that show promise in diagnosis, prevention, or treatment of rare diseases. VCA-894A received the Orphan Drug Designation in June 2023.
Reference
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 13, 2025. https://bit.ly/40a1f3A
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