FDA Grants Orphan Drug Designation to VGT-1849B for Polycythemia Vera Treatment

Vanda Pharmaceuticals has announced that the FDA has granted Orphan Drug Designation for VGT-1849B, a selective peptide nucleic acid-based JAK2 inhibitor for the treatment of polycythemia vera (PV). PV is a chronic myeloproliferative disorder characterized by aberrant hematopoiesis of myeloid lineage with exuberant red cell production and increased release of pro-inflammatory cytokines. The prevalence of PV in the US is estimated to affect 44 to 57 per 100,000 people. VGT-1849B utilizes a novel backbone chemistry, OliPass Peptide Nucleic Acid (OPNA), to target the JAK2 V617F gain-of-function mutation.

Title: Vanda Pharmaceuticals Secures FDA Orphan Drug Designation for Novel JAK2 Inhibitor VGT-1849B

Vanda Pharmaceuticals (Nasdaq: VNDA) has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VGT-1849B, a selective peptide nucleic acid-based JAK2 inhibitor for the treatment of polycythemia vera (PV). This designation is a significant milestone for Vanda, as it provides important development incentives to address this rare blood disorder affecting approximately 44 to 57 per 100,000 Americans [1].

PV is a chronic myeloproliferative disorder characterized by aberrant hematopoiesis of myeloid lineage, leading to exuberant red cell production and increased release of pro-inflammatory cytokines. More than 95% of PV patients harbor the JAK2 V617F gain-of-function mutation, which drives the disease [2].

VGT-1849B utilizes a novel backbone chemistry called OliPass Peptide Nucleic Acid (OPNA) to target the JAK2 V617F mutation. Unlike existing JAK inhibitors such as Jakafi®, Inrebic®, Ojjaara®, and Vonjo®, which lack selectivity for JAK2, VGT-1849B specifically targets JAK2 mRNA. This selectivity could offer improved safety and less frequent dosing compared to current treatments [1].

The FDA Orphan Drug Designation provides significant advantages, including tax credits, market exclusivity, and reduced regulatory fees. If approved, VGT-1849B could offer targeted efficacy with an improved safety profile and convenient infrequent dosing. This designation enhances Vanda's position in developing treatments for orphan hematological conditions [2].

Vanda Pharmaceuticals is a leading global biopharmaceutical company focused on developing innovative therapies to address high unmet medical needs. The company's commitment to addressing rare diseases underscores its dedication to improving patient outcomes [1].

References
[1] https://www.stocktitan.net/news/VNDA/vanda-pharmaceuticals-announces-fda-granted-orphan-drug-designation-2hbu489m1w7t.html
[2] https://www.marketscreener.com/news/vanda-pharmaceuticals-announces-fda-granted-orphan-drug-designation-for-vgt-1849b-a-novel-and-selec-ce7c50dfde8af122