Rare Disorder Gets First Approved Treatment

FDA approves Ryplazim (plasminogen, human-tvmh) to treat hypoplasminogenemia

(RxWiki News) The US Food and Drug Administration (FDA) has approved the first-ever treatment for a rare genetic disorder called plasminogen deficiency.

This treatment is called Ryplazim (plasminogen, human-tvmh). The FDA approved it to treat plasminogen deficiency type 1 in adults and children.

Plasminogen deficiency type 1, also called hypoplasminogenemia, can impair organ and tissue function and lead to blindness.

“Until now, there were no FDA-approved treatment options for patients with plasminogen deficiency type 1,” said Dr. Peter Marks, director of FDA’s Center for Biologics Evaluation and Research, in a press release. “Today’s approval helps address an unmet medical need for individuals affected by this rare genetic disease.”

People who have plasminogen deficiency do not have enough of a protein called plasminogen in their bodies. This protein helps the body break down clots of a substance called fibrin. Without enough plasminogen in the body, fibrin builds up. This fibrin buildup can cause lesions that affect tissue and organ function. And when these lesions occur in the eyes, they can lead to blindness.

Ryplazim's active ingredient is plasminogen. According to the FDA, taking this newly approved medication increases the amount of plasminogen in the blood. This increase in plasminogen can allow the body to break down fibrin and prevent harmful fibrin lesions.

The FDA approved Ryplazim based on the results of a trial of 15 adults and children who had plasminogen deficiency type 1. All of the study patients received Ryplazim every two to four days for 48 weeks.

Eleven patients had fibrin lesions at the start of the study. By the end of the study, all of those patients had seen at least 50 percent improvement in their lesions. For the four patients who did not have lesions at the start of the study, Ryplazim appeared to completely prevent lesions from forming.

Side effects of Ryplazim included stomach pain, nausea, bloating, limb pain, bleeding, constipation, fatigue, headache, dry mouth, joint pain, back pain and dizziness.

The FDA approved this drug for ProMetic Biotherapeutics Inc.

Talk to your health care provider about the best treatment for your condition.

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Review Date: 
June 17, 2021
Last Updated:
June 21, 2021