Off-Label Uses of Nemolizumab: A Narrative Review.

BACKGROUND: Nemolizumab, a humanized monoclonal antibody targeting interleukin-31 receptor α (IL-31Rα), is approved for atopic dermatitis (AD) and prurigo nodularis (PN) in adults and pediatric patients 12 years and older, following phase 3 trials demonstrating significant reductions in pruritus and disease severity in patients inadequately controlled with topical therapies. Emerging evidence has demonstrated rapid and sustained pruritus reduction in diverse pruritic conditions beyond its approved indications.

METHODS: We reviewed the literature from 2021 to 2025 examining off label nemolizumab use, analyzing case reports, case series, randomized controlled trials, and meta-analyses across dermatologic and systemic pruritic conditions.

RESULTS: Across 16 studies with diverse diagnoses, nemolizumab demonstrated consistent efficacy in multiple off-label conditions including cutaneous amyloidosis, lichen simplex chronicus, uremic and dialysis-associated pruritus, cholestatic pruritus, and refractory dermatologic conditions. Adverse events were consistent with those described in its package insert.

CONCLUSIONS: Current evidence supports nemolizumab as a promising therapeutic option for treatment-refractory pruritic conditions. The rapid onset, favorable tolerability, and broad efficacy across conditions with elevated IL-31 signaling suggest significant clinical utility, though larger randomized trials are needed for definitive recommendations. &nbsp.