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Uveitis, a leading cause of preventable blindness around the world, is a critically underserved disease in regard to the medications approved for use. Multiple immunomodulatory therapy (IMT) drugs are appropriate for uveitis therapy but are still off-label. These IMT agents, including antimetabolites, calcineurin inhibitors, alkylating agents, and biologic agents, have been designated as "orphan drugs" and are widely used for systemic autoimmune diseases or organ transplantation.

A review and update on orphan drugs for the treatment of noninfectious uveitis