Vanda Pharmaceuticals Inc. (VNDA) announced on Friday that the U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to VGT-1849A. This compound is a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor intended for the treatment of polycythemia vera (PV).
The Orphan Drug Designation is provided by the FDA to investigational therapies aimed at treating rare medical conditions and offers benefits to drug developers.
Polycythemia vera is a rare hematologic malignancy, affecting approximately 1 in every 2,000 Americans.
This chronic myeloproliferative disorder is marked by abnormal hematopoiesis of the myeloid lineage, characterized by excessive red blood cell production and an increased release of pro-inflammatory cytokines. Over 95% of individuals with PV possess the JAK2 V617F gain-of-function mutation, which results in abnormal JAK2 production.
