FDA approves new treatment for patients with hypoplasminogen

FDA approves new treatment for patients with hypoplasminogenemia


FDA approves new treatment for patients with hypoplasminogenemia
Today, the U.S. Food and Drug Administration approved Ryplazim (plasminogen, human-tmvh) for the treatment of patients with plasminogen deficiency type 1, also referred to as hypoplasminogenemia, a disorder that can impair normal tissue and organ function and may lead to blindness.
Until now, there were no FDA-approved treatment options for patients with plasminogen deficiency type 1. Today's approval helps address an unmet medical need for individuals affected by this rare genetic disease."
Peter Marks, M.D., Ph.D., Director, FDA's Center for Biologics Evaluation and Research
Individuals with this disease lack a protein called plasminogen, which is responsible for the ability of the body to break down fibrin clots. Plasminogen deficiency leads to an accumulation of fibrin, causing the development of growths (lesions) that can impair normal tissue and organ function and may lead to blindness when these lesions affect the eyes.

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