FDA Approves Treatment for Plasminogen Deficiency Type 1

FDA announced on June 4, 2021, that it has approved Ryplazim (plasminogen, human-tmvh) to treat plasminogen deficiency type 1. Also known as hypoplasminogenemia, the rare disorder can impair tissue and organ function and may lead to blindness, according to the agency. Ryplazim was granted Orphan Drug and Fast Track designations as well as Priority Review and a Rare Pediatric Disease Priority Review Voucher.

Ryplazim’s active ingredient, plasminogen, is purified from human plasma. Ryplazim helps increase the level of plasminogen in patients who lack the protein, the agency says, “enabling a temporary correction of the plasminogen deficiency and reduction or resolution of the lesions.”

FDA based the safety and effectiveness of the drug on one single-arm, open-label clinical trial that included 15 adult and pediatric patients with plasminogen deficiency type 1. Patients received the drug every two to four days for 48 weeks. A 50% improvement of lesions was seen in all 11 patients who had lesions at baseline. No recurrent or new lesions were seen in any of the 15 patients through the time of treatment.

“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. “Today’s approval helps address an unmet medical need for individuals affected by this rare genetic disease.”

Source: FDA