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Vanda Pharmaceuticals' Imsidolimab Receives Orphan Drug Designation from EU

Vanda Pharmaceuticals announced that its investigational drug imsidolimab has received Orphan Designation from the European Commission, based on the positive opinion received from the European Medicines Agency's, or EMA, Committee for Orphan Medicinal Products, or COMP, for the treatment of Generalized Pustular Psoriasis, or GPP, which is a severe, chronic, and potentially life-threatening inflammatory skin disease driven by dysregulation in the interleukin-36 signaling pathway. The FDA and Japan's Ministry of Health, Labour and Welfare have previously granted Orphan Drug Designation to imsidolimab for the treatment of GPP. This marks the first time the European Commission has granted orphan designation recognition for a drug to treat GPP in the European Union. GPP is clinically distinct from plaque psoriasis and is characterized 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. This designation follows similar regulatory recognitions in the U.S. and Japan.

Vanda Pharmaceuticals Receives EU Orphan Drug Designation

Vanda Pharmaceuticals announced that the Committee for Orphan Medicinal Products at the European Medicines Agency has adopted a positive opinion recommending orphan drug designation for imsidolimab, the company's investigational medicinal product, a high-affinity humanized immunoglobulin G4 monoclonal antibody, for the treatment of generalized pustular psoriasis. This marks the first time the EMA has granted orphan drug designation recognition for a drug to treat GPP in the European Union. 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 signaling pathway. It is characterized by widespread pustular eruptions, systemic inflammation, and serious complications that can lead to increased mortality. "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 for GPP is currently under review by the FDA with a target action date of December 12, 2026.

Vanda Pharmaceuticals Receives FDA Rare Pediatric Disease Designation for VCA-894A

The company states: "Vanda Pharmaceuticals announced that the FDA has granted Rare Pediatric Disease Designation to VCA-894A, Vanda's investigational antisense oligonucleotide therapy for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), a rare, serious, and progressive inherited neurological disorder. The designation was granted by the FDA's Office of Orphan Products Development and Office of Pediatric Therapeutics. VCA-894A is being developed for a patient who was first diagnosed at an early age with a rare subtype of Charcot-Marie-Tooth disease known as CMT2S.1 CMT2S is an inherited neuromuscular disorder that progressively leads to muscle weakness and loss of motor function, and has an estimated prevalence of less than 1 in 1,000,000 worldwide.2 VCA-894A's therapeutic target is a unique variant of CMT2S not yet observed in any other patient. The severity and clinical presentations of CMT2S are influenced by the diverse genetic variants associated with CMT disease. The FDA determined that CMT2S qualifies as a rare pediatric disease because it is a serious or life-threatening condition whose manifestations primarily affect individuals from birth through 18 years of age and it meets the statutory definition of a rare disease."