Biopharmaceutical company Vanda Pharmaceuticals Inc. (Nasdaq VNDA) announced on Friday that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor aimed at treating polycythaemia vera (PV), a rare hematologic malignancy affecting approximately 1 in 2000 Americans.

PV is a chronic myeloproliferative disorder marked by excessive red blood cell production and increased pro-inflammatory cytokines, driven by the JAK2 V617F mutation in over 95% of patients. VGT-1849A selectively targets JAK2, reducing JAK2V617F-driven pathogenic signaling and suppressing the malignant proliferation of hematopoietic cells. Unlike existing JAK2 inhibitors, VGT-1849A offers specificity for JAK2, potentially minimizing off-target effects and reducing the risk of adverse side effects seen with broader JAK inhibition. The selective targeting of JAK2 aims to reduce infections and toxicities associated with inhibitors that also block other JAK family members.

Vanda's development of VGT-1849A represents a potential advancement in treating PV with an improved safety profile. If approved, VGT-1849A could offer a more effective, safer treatment option for PV and other JAK2-driven hematologic malignancies.