The U.S. Food and Drug Administration (FDA) has granted approval to Prometic Biotherapeutics for plasminogen (brand name: Ryplazim) as a treatment for plasminogen deficiency type 1. This is the first FDA approved treatment for this condition.
Plasminogen deficiency type 1, also known as hypoplasminogenemia, is a rare genetic condition due to mutations of the plasminogen gene.
Plasminogen deficiency is associated with inflamed growths on the mucous membranes which can impair normal tissue and/or organ function. The area most commonly affected is the conjunctiva which often leads to ligneous conjunctivitis. This in turn can lead to blindness.
The FDA approval was largely based on the positive results from a single-arm, open-label clinical trial of the effectiveness and safety of plasminogen. The clinical trial enrolled 15 adult and pediatric patients with plasminogen deficiency type 1, all of whom received plasminogen every two to four days for 48 weeks. Of the 11 participants who had lesions at baseline, all experienced a 50% or more improvement of their lesions, and there was an absence of recurrent or new lesions in all 15 participants throughout the 48 treatment period.
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