Posted by Michael Wonder on 04 Jun 2021
FDA approves first treatment for patients with plasminogen deficiency, a rare genetic disorder
4 June 2021 - Today, the U.S. FDA 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.
The effectiveness and safety of Ryplazim is primarily based on one single-arm, open-label clinical trial enrolling 15 adult and paediatric patients with plasminogen deficiency type 1.
