Vanda Pharmaceuticals recently announced that its investigational drug imsidolimab has received Orphan Drug Designation (ODD) from the European Commission (EC), based on the positive opinion received from the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP), for the treatment of Generalised Pustular Psoriasis (GPP), which is a severe, chronic, and potentially life-threatening inflammatory skin disease driven by dysregulation in the interleukin-36 (IL-36) signaling pathway.

The US Food and Drug Administration (FDA) and Japan's Ministry of Health, Labour and Welfare (MHLW) have previously granted ODD to imsidolimab for the treatment of GPP.

This marks the first time EC has granted orphan designation recognition for a drug to treat GPP in the EU. GPP is clinically distinct from plaque psoriasis and is characterised by widespread pustular eruptions, systemic inflammation, and serious complications that can lead to increased mortality. Imsidolimab inhibits IL-36 receptor signaling, addressing the deficiency in the endogenous IL-36 receptor antagonist commonly observed in patients with GPP.

Dr. Mihael H. Polymeropoulos, President, CEO and Chairman of the Board, Vanda Pharma, said, "The European Commission's Orphan Designation for imsidolimab represents an important milestone for the GPP community and for our efforts to bring this investigational therapy to patients in Europe. With orphan designations now received in Europe, the US and Japan, we remain focused on advancing imsidolimab for patients in these markets who need new treatment options."

This designation follows similar regulatory recognitions in the US and Japan. Additionally, the imsidolimab Biologics License Application (BLA) for GPP is currently under review by the FDA with a target action date of December 12, 2026.