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News From the Food and Drug Administration
August 3, 2021

First Treatment for Plasminogen Deficiency Is Approved

JAMA. 2021;326(5):379. doi:10.1001/jama.2021.12278

Purified plasminogen that’s derived from human plasma has become the first FDA-approved treatment for plasminogen deficiency type 1. Plasminogen, human-tvmh, marketed as Ryplazim, is administered intravenously every 2 to 4 days.

Plasminogen deficiency type 1 results from PLG gene variants that impair plasminogen production, function, or both. Without sufficient plasminogen, the body can’t dissolve fibrin clots, which causes fibrin to accumulate. The excess leads to inflammation and growths that develop on mucous membranes. The growths can lead to vision loss, breathing problems, ulcers, or other problems, depending on where in the body they develop. Previously, treatment consisted of surgically removing the lesions and giving plasma infusions.