Vanda Pharmaceuticals stock rises after FDA orphan drug designation
Investing.com -- Vanda Pharmaceuticals (NASDAQ:VNDA) stock rose 6% after the company announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its VGT-1849B treatment for polycythemia vera (PV).
VGT-1849B is a selective peptide nucleic acid-based JAK2 inhibitor designed to target the treatment of PV, a chronic myeloproliferative disorder that affects an estimated 44 to 57 per 100,000 people in the United States. The condition is characterized by excessive red blood cell production and increased release of pro-inflammatory cytokines.
The company’s treatment approach differs from existing JAK inhibitors on the market by specifically targeting JAK2 mRNA with high precision, potentially reducing off-target effects that can lead to increased toxicity. More than 95% of PV patients harbor the JAK2 V617F gain-of-function mutation that leads to aberrant JAK2 production.
Currently available JAK inhibitors such as Jakafi, Inrebic, Ojjaara, and Vonjo are not solely selective to JAK2, which can result in binding to off-target kinases. Vanda’s approach aims to reduce JAK2 protein production without affecting other JAK family members, potentially offering a more favorable safety profile.
VGT-1849B utilizes a novel backbone chemistry called OliPass Peptide Nucleic Acid that improves cell permeability and affinity for RNA by introducing cationic lipid moieties onto nucleobases.
The Orphan Drug Designation provides various benefits to drug developers addressing rare medical conditions, including tax credits for clinical trials and exemption from user fees.
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