Vanda Pharmaceuticals Announces EMA Positive Opinion for Orphan Drug Designation of Imsidolimab for Generalized Pustular Psoriasis – A Milestone Recognition of GPP as a Distinct Rare Disease in Europe

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Vanda Pharmaceuticals Announces EMA Positive Opinion for Orphan Drug Designation of Imsidolimab for Generalized Pustular Psoriasis – A Milestone Recognition of GPP as a Distinct Rare Disease in Europe

PR Newswire
Fri, July 17, 2026 at 2:00 AM GMT+5:30
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WASHINGTON, July 16, 2026 /PRNewswire/ — Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the Committee for Orphan Medicinal Products at the European Medicines Agency (EMA) has adopted a positive opinion recommending orphan drug designation for imsidolimab, the company’s investigational medicinal product, a high-affinity humanized immunoglobulin G4 (IgG4) monoclonal antibody, for the treatment of generalized pustular psoriasis (GPP)

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

“The EMA’s positive opinion is a significant milestone that reinforces the highly encouraging clinical data supporting the imsidolimab program and formally recognizes GPP as an orphan disease in the EU for the first time,” said Dr. Mihael H. Polymeropoulos, “This designation highlights the urgent unmet medical need in GPP and brings us one step closer to delivering a meaningful new therapy to patients in the EU.”

This designation follows similar regulatory recognitions in the United States 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

1. Smieszek, S. et al. Efficacy and Safety of Imsidolimab for Generalized Pustular Psoriasis. NEJM Evidence 5, (2026)

2. Ministry of Health Labour and Welfare (MHLW), “Pustular psoriasis (generalized type) (Designated Intractable Disease 37),” Japan Intractable Diseases Information Center. Accessed: Mar. 10, 2026. [Online]. Available: https://www.nanbyou.or.jp/entry/168

3. H. Fujita, R. Iwasaki, S. Tsuboi, Y. Murashiuma, and M. Akiyama, “Regional differences in the prevalence of generalized pustular psoriasis in Japan,” J. Dermatol., vol. 51, no. 3, pp. 380–390, Mar. 2024, doi: 10.1111/1346-8138.17089

4. H. Miyachi et al., “Treatments and outcomes of generalized pustular psoriasis: A cohort of 1516 patients in a nationwide inpatient database in Japan,” J. Am. Acad. Dermatol., vol. 86, no. 6, pp. 1266–1274, Jun. 2022, doi: 10.1016/j.jaad.2021.06.008