This marks the first time the EMA has granted orphan drug designation recognition for a drug to treat GPP in the European Union (EU). GPP is a severe, chronic, and potentially life-threatening inflammatory skin disease, genetically and clinically distinct from plaque psoriasis, driven by dysregulation in the interleukin-36 (IL-36) signaling pathway.1 It is characterized by widespread pustular eruptions, systemic inflammation, and serious complications that can lead to increased mortality.234

Imsidolimab inhibits IL-36 receptor signaling, addressing the deficiency in the endogenous IL-36 receptor antagonist commonly observed in patients with GPP.

The EMA's orphan drug designation is granted to medicines intended for the treatment of life-threatening or chronically debilitating rare conditions affecting fewer than 5 in 10,000 people in the EU. Benefits include protocol assistance, reduced regulatory fees, and market exclusivity provisions in the EU following approval.