By VETTAPHARMA reporter – Derek Roche: Vanda Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the filing of its Biologics License Application (BLA) for imsidolimab for the treatment of Generalized Pustular Psoriasis (GPP), with a target action date of December 12, 2026.
GPP is a rare, chronic, life-threatening autoinflammatory skin disorder characterized by sudden flares of widespread pustules, erythema and systemic symptoms such as fever and fatigue. The disease is genetically characterized under OMIM #614204, and its molecular etiology is mainly attributed to excessive activity of the interleukin-36 (IL-36) pathway. The majority of cases with an identified single gene defect are caused by genetic variants in the IL36RN gene, which encodes the IL-36 receptor antagonist.
Imsidolimab is a fully humanized IgG4 monoclonal antibody that inhibits IL-36 receptor signaling and is designed to address the underlying inflammatory mechanism in GPP where IL-36 signaling is unbalanced. If approved, the therapy could address a significant unmet medical need and offer potential benefits over existing treatments.
The drug was evaluated in global clinical studies conducted in the United States, France, Spain, Poland, Turkey, Malaysia, Thailand, Georgia, Tunisia, Taiwan and Morocco. In the pivotal GEMINI-1 and GEMINI-2 efficacy studies, a single intravenous dose of imsidolimab resulted in 53% of patients achieving clear or almost clear skin (GPPPGA 0/1) at Week 4, compared to 13% on placebo. Efficacy was maintained over an approximately 2-year maintenance period with monthly dosing, during which no flares occurred in the active treatment arm. The therapy demonstrated a favorable safety profile and a low incidence of anti-drug antibodies.
GPP prevalence estimates vary widely by region, ranging from approximately 2 to 124 cases per million worldwide, with lower prevalence reported in Europe and higher rates in parts of Asia.
Vanda noted that, if approved, imsidolimab would become the third new drug product approved for the company in the past 12 months, following NEREUS™ (tradipitant) and BYSANTI™ (milsaperidone). Regulatory and patent exclusivity for imsidolimab is expected to extend into the late 2030s. Vanda holds an exclusive global license for the development and commercialization of imsidolimab from AnaptysBio (Nasdaq: ANAB).
