Vanda Pharmaceuticals Receives Orphan Drug Designation for VGT-1849B to Treat Polycythemia Vera

Vanda Pharmaceuticals announced that the FDA has granted orphan drug designation for VGT-1849B, a novel JAK2 inhibitor for the treatment of polycythemia vera. The drug targets the JAK2 V617F mutation, which causes the disease, and aims to reduce red blood cell production and alleviate symptoms. VGT-1849B has shown promise in preclinical trials and could offer targeted efficacy with an improved safety profile and convenient infrequent dosing if approved. Orphan drug designation provides benefits to drug developers, including tax credits and market exclusivity.

Vanda Pharmaceuticals (Nasdaq: VNDA) has received a significant milestone in its efforts to develop innovative treatments for rare diseases. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to VGT-1849B, a novel JAK2 inhibitor for the treatment of polycythemia vera (PV).

Polycythemia vera is a rare blood disorder affecting approximately 44-57 per 100,000 people in the United States. The condition is characterized by an overproduction of red blood cells, leading to an increased risk of blood clots and other complications. The FDA's Orphan Drug Designation provides important development incentives for addressing this rare medical condition, including tax credits and market exclusivity [1].

VGT-1849B is a selective peptide nucleic acid-based JAK2 inhibitor that specifically targets JAK2 mRNA, distinguishing it from existing treatments like Jakafi®, Inrebic®, Ojjaara®, and Vonjo®. The drug's unique OPNA backbone chemistry enhances cell permeability and RNA affinity, potentially offering improved safety and less frequent dosing compared to current treatments. By precisely targeting JAK2 mRNA, VGT-1849B could theoretically maintain efficacy while reducing adverse effects seen with pan-JAK inhibitors, particularly infection risks [2].

The selective nature of VGT-1849B addresses a significant limitation of current therapies. Existing JAK inhibitors lack selectivity for JAK2, leading to off-target effects on other kinases that can cause immunosuppression and other toxicities. By specifically targeting JAK2 mRNA, VGT-1849B could potentially reduce the risk of these adverse effects and improve patient outcomes.

Vanda Pharmaceuticals has achieved a significant regulatory milestone with the FDA granting Orphan Drug Designation for VGT-1849B. This designation enhances Vanda's position in developing treatments for orphan hematological conditions. The technology behind VGT-1849B represents a potentially important advancement in targeting precision. Unlike existing JAK inhibitors, VGT-1849B employs an antisense oligonucleotide approach with OliPass Peptide Nucleic Acid (OPNA) technology to specifically target JAK2 mRNA [1].

If approved, VGT-1849B could offer targeted efficacy with an improved safety profile and convenient infrequent dosing. This regulatory milestone enhances Vanda's position in developing treatments for orphan hematological conditions.

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