Plasminogen Replacement Therapy Gets FDA Rare Pediatric Disease Designation

Orphan drug and fast-track status underscore significant unmet need for therapy

The FDA has granted a rare pediatric disease designation to Prometic Life Sciences’ Ryplazimä, a plasminogen replacement therapy for the treatment of patients with congenital plasminogen deficiency. In addition to the rare pediatric disease designation, Ryplazimä has already been granted FDA orphan drug and fast-track status.

Rare pediatric disease designations are given to drugs that treat serious or life-threatening diseases in which the manifestations primarily affect individuals up to 18 years of age, including neonates, infants, children, and adolescents. If approved, Ryplazimä will be eligible to receive a rare pediatric disease priority review voucher.

Plasminogen is a naturally occurring protein that is synthesized by the liver and circulates in the blood. Activated plasminogen, plasmin, is a fundamental component of the fibrinolytic system and is the main enzyme involved in the lysis of blood clots and clearance of extravasated fibrin. Plasminogen is therefore vital in wound healing, cell migration, tissue remodeling, angiogenesis, and embryogenesis.

The most common condition associated with plasminogen deficiency is ligneous conjunctivitis, which is characterized by thick, woody (ligneous) growths on the conjunctiva of the eye, and if left untreated, can lead to corneal damage and blindness. Ligneous growths tend to recur after surgical excision, thereby requiring multiple surgeries.

While ligneous conjunctivitis is the best-characterized lesion of plasminogen deficiency, hypoplasminogenemia is a multisystemic disease that can also affect the ears, sinuses, tracheobronchial tree, genitourinary tract, and gingiva. Tracheobronchial lesions, including hyperviscous secretions, can result in respiratory failure. Hydrocephalus has also been reported in children with severe hypoplasminogenemia, apparently related to the deposition of fibrin in the cerebral ventricular system.

Source: Prometic; August 29, 2017.