FDA Approves First Ever Treatment for Plasminogen Deficiency Type 1

This weekend, the US Food and Drug Administration (FDA) approved plasminogen, human-tmvh (Ryplazim) for plasminogen deficiency type 1. This greenlight marks the first time a treatment has been approved for the rare genetic disease, a cause of impairment of organ function and tissue as well as blindness.

Patients with plasminogen deficiency type 1, or hypolasminogenemia, suffer from an inability to break down fibrin clots. As such, the accumulation of fibrin can lead to lesion growths, thus affecting normal tissue and organ function.

Plasminogen, human-tmvh thus provides temporary relief by inducing an increase in the plasma level of plasminogen, the protein responsible for the breaking down of fibrin.

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

Supporting data

The FDA approval was supported by a single-arm, open-label clinical trial that involved 15 adult and pediatric patients with plasminogen deficiency type 1.

Patients received 6.6 mg/kg of treatment every 2-4 days over the course of 48 weeks. All patients with lesions at baseline (n = 11) demonstrated ≥50 improvement.

Further, any of the 15 patients saw an absence of recurrent or new lesions through the 48-week treatment period.

The most common adverse events associated with the plasminogen, human-tmvh were abdominal pain, bloating, nausea, bleeding, limb pain, fatigue, constipation, dry mouth, headache, dizziness, joint pain, and back pain.

Approval was granted to Liminal BioSciences.