Vanda Pharmaceuticals said the European Medicines Agency’s Committee for Orphan Medicinal Products has issued a positive opinion recommending orphan drug designation for imsidolimab for the treatment of generalized pustular psoriasis (GPP).
Imsidolimab is a high‑affinity humanized IgG4 monoclonal antibody that targets interleukin‑36 (IL‑36) receptor signaling. Dysregulation of the IL‑36 pathway and deficiency of the endogenous IL‑36 receptor antagonist are commonly observed in patients with GPP, a severe and potentially life‑threatening inflammatory skin disease that is genetically and clinically distinct from plaque psoriasis.
According to Vanda, this is the first time a medicine for GPP has received an orphan drug designation recommendation in the European Union. GPP is characterized by widespread pustular skin eruptions, systemic inflammation, and complications that can increase mortality risk. As a rare condition, it is estimated to affect fewer than 5 in 10,000 people in the EU.
Orphan drug designation in the EU provides incentives such as protocol assistance, reduced regulatory fees, and a period of market exclusivity if the product is approved. Vanda said the decision underscores both the unmet medical need in GPP and regulatory recognition of the disease as a distinct orphan indication in Europe.
The EMA opinion follows similar rare-disease designations for imsidolimab in the United States and Japan. In the U.S., a biologics license application for imsidolimab in GPP is under review by the Food and Drug Administration, with a target action date of December 12, 2026.
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