PROMETIC RECEIVES RARE PEDIATRIC DISEASE DESIGNATION FROM U.S. FDA FOR ITS PLASMINOGEN REPLACEMENT THERAPY
- Triple designation of orphan drug, fast track status, combined with rare pediatric designation, underscores significant unmet need for therapy
' LAVAL, QUEBEC, CANADA – Aug. 29, 2017 – Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (Prometic) today announced that the U.S Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation to Prometic’s 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 Orphan Drug and Fast Track Designation by the FDA.
The FDA grants Rare Pediatric Disease Designation for serious or life-threatening diseases in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents. If approved, Prometic’s plasminogen replacement therapy will be eligible to receive a rare pediatric disease priority review voucher.
“We are pleased that RyplazimTM has received this pediatric designation from the FDA. In addition to Ryplazim’sTM orphan drug status, this designation, represents another validation of the approach we have taken regarding our plasminogen’s clinical program,” said Mr. Pierre Laurin, President and Chief Executive Officer of Prometic. “The pediatric designation also provides an important incentive for Prometic to continue developing therapies addressing unmet medical needs for children with rare diseases. We look forward to continue to work closely with the FDA to bringing this innovative therapy to patients suffering from plasminogen deficiency.”
About Plasminogen
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.
About Plasminogen Deficiency
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 multi-systemic disease that can also affect the ears, sinuses, tracheobronchial tree, genitourinary tract, and gingiva. Tracheobronchial lesions including hyper viscous 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.'
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