WASHINGTON, DC — Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for imsidolimab, a novel IgG4 IL-36 receptor antagonist, to treat generalized pustular psoriasis (GPP). Imsidolimab inhibits IL-36 receptor signaling, addressing the deficiency in the endogenous IL-36RA regulator commonly seen in GPP patients due to IL36RN gene mutations.
The BLA is supported by positive results from the global Phase 3 GEMINI-1 and GEMINI-2 studies, where a single intravenous dose of imsidolimab led to rapid disease clearance, achieving clear or almost clear skin, with efficacy maintained throughout an approximately 2-year maintenance study period with monthly doses. Imsidolimab demonstrated a favorable safety profile with no clinically meaningful safety signals.
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. Driven primarily by loss-of-function mutations in IL36RN, GPP represents a significant unmet medical need, with prevalence estimates varying widely by region, ranging from approximately 2 to 124 cases per million worldwide (e.g., lower in Europe and higher in parts of Asia).1,2,3,4
“The submission of the BLA for imsidolimab marks a critical milestone in our efforts to bring this innovative therapy to patients suffering from GPP,” said Mihael H. Polymeropoulos, M.D., President, CEO and Chairman of the Board of Vanda Pharmaceuticals. “Imsidolimab builds on our growing expertise in rare orphan disorders and our anti-inflammatory portfolio, including Ponvory®, which is approved for the treatment of relapsing forms of multiple sclerosis and is in clinical development for the treatment of psoriasis and ulcerative colitis. We look forward to potential FDA approval and leveraging our commercial infrastructure to address this debilitating condition.”
Vanda has requested priority review for the BLA, citing GPP’s status as a rare orphan disease with significant unmet need. If granted, priority review would establish a six-month review cycle, with a potential FDA approval of imsidolimab for the treatment of GPP as early as mid-2026.
References
- Prinz, J. C. et al. Prevalence, comorbidities and mortality of generalized pustular psoriasis: A literature review. Journal of the European Academy of Dermatology and Venereology 37, 256–273 (2022).
- Marrakchi, S. et al. Interleukin-36–Receptor Antagonist Deficiency and Generalized Pustular Psoriasis. New England Journal of Medicine 365, 620–628 (2011).
- Sugiura, K. et al. The Majority of Generalized Pustular Psoriasis without Psoriasis Vulgaris Is Caused by Deficiency of Interleukin-36 Receptor Antagonist. Journal of Investigative Dermatology 133, 2514–2521 (2013).
- Sachen, K. L., Arnold Greving, C. N. & Towne, J. E. Role of IL-36 cytokines in psoriasis and other inflammatory skin conditions. Cytokine 156, 155897 (2022).
About GEMINI-1 and GEMINI-2 Studies
In the 45-patient GEMINI-1 Phase 3 trial, patients were randomized 1:1:1 to a single intravenous infusion of 750 mg imsidolimab, 300 mg imsidolimab, or placebo on Day 0. At Week 4 (primary endpoint), 53% of patients receiving either dose of imsidolimab achieved a GPPGA score of 0/1 (clear or almost clear skin), compared to 13% on placebo (p=0.0131 for 750 mg dose).
Responders from GEMINI-1 were re-randomized in GEMINI-2 to monthly subcutaneous maintenance dosing with 200 mg imsidolimab or placebo, followed for up to 116 weeks. All patients on active maintenance therapy maintained clear or almost clear skin with no flares, compared to 25% maintenance and 63% flare rate in the placebo group. Across both studies, imsidolimab demonstrated a favorable safety profile, with no treatment-related serious adverse events or discontinuations due to adverse events.
About Imsidolimab and GPP
Imsidolimab is a fully humanized IgG4 monoclonal antibody that inhibits IL-36 receptor signaling and is being developed for GPP, a rare orphan indication. 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).
GPP flares involve painful pustules over large skin areas, accompanied by redness, itching, and systemic symptoms, and can be life-threatening if untreated.
About Vanda Pharmaceuticals Inc.
Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients. For more on Vanda Pharmaceuticals Inc., please visit www.vandapharma.com and follow us on X.
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