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Plasminogen is a plasma-derived biologic medication used for the treatment of type 1 plasminogen deficiency (hypoplasminogenemia), a rare genetic disorder that can impair normal tissue and organ function and may lead to blindness[2][3][5]. Plasminogen is a pro-enzyme (zymogen) produced in the liver and circulates in the blood. It is converted into its active form, plasmin, by tissue plasminogen activator (tPA) or urokinase-like activator (uPA). Plasmin acts as a fibrinolytic enzyme that breaks down fibrin clots as part of the body's natural clot resolution process[2][6]. The drug increases circulating levels of functional plasminogen to promote healing of lesions associated with deficiency. Ryplazim was developed specifically for intravenous administration to patients with confirmed type 1 deficiency[5]. The U.S. FDA has approved Ryplazim for this indication; it received orphan drug designation, fast track status, priority review, and a rare pediatric disease priority review voucher[5].
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