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ADX-2191 granted orphan drug designation by the EMA for the treatment of primary LBCL of immune-privileged sites

By Dylan Barrett

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Sep 2, 2025

Learning objective: After reading this article, learners will be able to cite a new clinical development in the treatment of large B-cell lymphoma.


On August 28, 2025, the European Medicines Agency granted orphan drug designation to ADX-2191, a sterile, non-compounded intravitreal formulation of methotrexate, for the treatment of patients with primary large B-cell lymphoma of immune-privileged sites including primary vitreoretinal lymphoma, for which there are currently no approved therapies in the European Union.1 ADX-2191 was previously granted orphan drug designation by the U.S. Food and Drug Administration for the treatment of patients with primary vitreoretinal lymphoma.1 

A proposed clinical trial will assess cancer cell clearance after 30 days of therapy in up to 20 patients following 1:1 randomization to receive either one or eight intraocular injections of ADX-2191. The trial is expected to begin in the second half of 2025.1 

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