Vanda Pharmaceuticals (NASDAQ:VNDA) shares climbed 6% after the company revealed that the U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to its investigational therapy VGT-1849B for the treatment of polycythemia vera (PV).
VGT-1849B is a selective peptide nucleic acid-based JAK2 inhibitor aimed at treating PV, a chronic blood disorder that affects roughly 44 to 57 individuals per 100,000 in the U.S. The disease is marked by excessive red blood cell production and heightened levels of pro-inflammatory cytokines.
Unlike existing JAK inhibitors, Vanda’s candidate is engineered to precisely target JAK2 mRNA, which could reduce off-target activity and minimize toxicity risks. More than 95% of patients with PV carry the JAK2 V617F mutation, which drives abnormal JAK2 protein production.
Currently marketed JAK inhibitors such as Jakafi, Inrebic, Ojjaara, and Vonjo lack strict selectivity for JAK2 and may bind to other kinases, raising safety concerns. Vanda’s strategy is designed to suppress JAK2 protein synthesis without interfering with other JAK family members, potentially offering a safer treatment profile.
The therapy incorporates OliPass Peptide Nucleic Acid technology, which enhances cellular permeability and RNA binding by adding cationic lipid groups to nucleobases.
Receiving Orphan Drug Designation gives Vanda several development incentives, including clinical trial tax credits and exemption from certain FDA fees, providing a pathway to accelerate its PV program.
Vanda Pharmaceuticals stock price
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