FDA Approves First Treatment for Patients with Plasminogen D
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.

Individuals with this disease lack a protein called plasminogen. Plasminogen deficiency leads to an accumulation of fibrin, causing the development of growths that can impair normal tissue and organ function and may lead to blindness when these lesions affect the eyes. The active ingredient in Ryplazim is plasminogen, purified from human plasma. Treatment with Ryplazim helps to increase the plasma level of plasminogen - enabling a temporary correction of the plasminogen deficiency and reduction or resolution of the lesions.

The effectiveness and safety of Ryplazim is primarily based on one single-arm, open-label (unblinded) clinical trial enrolling 15 adult and pediatric patients with plasminogen deficiency type 1. All patients received Ryplazim administered every two to four days for 48 weeks. The effectiveness of Ryplazim was demonstrated by at least 50% improvement of their lesions in all 11 patients who had lesions at baseline, and absence of recurrent or new lesions in any of the 15 patients through the 48 weeks of treatment.

The most common side effects reported by patients who received Ryplazim were abdominal pain, bloating, nausea, bleeding, limb pain, fatigue, constipation, dry mouth, headache, dizziness, joint pain, and back pain. The FDA granted Ryplazim Orphan Drug designation, which provides incentives to assist and encourage drug development for rare diseases.

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