US pharma company Vanda Pharmaceuticals has announced that the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for imsidolimab, a novel therapy targeting generalized pustular psoriasis (GPP).

 

Imsidolimab, an IgG4 IL-36 receptor antagonist, works by inhibiting IL-36 receptor signaling, directly addressing the deficiency in the IL-36RA regulator common in GPP patients with IL36RN gene mutations.

 

The BLA follows positive results from the global Phase 3 GEMINI-1 and GEMINI-2 trials, where a single intravenous dose of imsidolimab led to rapid disease clearance. Patients achieved clear or nearly clear skin, with efficacy maintained over approximately two years through monthly dosing. The drug demonstrated a favorable safety profile with no clinically meaningful safety concerns.

 

GPP is a rare, chronic, and potentially life-threatening autoinflammatory skin disorder, marked by sudden flares of pustules, red skin, fever, and fatigue. Driven primarily by IL36RN gene mutations, the disease affects between 2 and 124 people per million worldwide, depending on the region.

 

“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, 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, citing GPP’s status as a rare orphan disease with urgent unmet needs. If granted, the FDA could complete its review in six months, potentially bringing imsidolimab to patients as early as mid-2026.