PROMETIC ANNOUNCES FDA ACCEPTANCE OF ITS BIOLOGICS LICENSE APPLICATION FOR PLASMINOGEN (RYPLAZIM™)
- Prescription Drug User Fee Act (PDUFA) action date is set for April 14, 2018
- Ryplazim™ previously granted Fast Track, Rare Pediatric Disease and Orphan Drug Designations by U.S. FDA
'Laval, Quebec, Canada – October 13, 2017 – Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (Prometic) today announced that the U.S Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for its plasminogen replacement therapy (RyplazimTM) having granted a priority review status and set a Prescription Drug User Fee Act (PDUFA) action date for April 14, 2018, Ryplazim™ had previously been granted Fast Track, Orphan Drug and Rare Pediatric Disease designations by the U.S. FDA.
"The acceptance of our BLA filing and setting of the PDUFA date by the FDA is a significant milestone for Prometic”, said Mr. Pierre Laurin, President and Chief Executive Officer of Prometic. “The FDA has recognized the seriousness and unmet need of available therapeutic solutions for plasminogen deficiency, granting orphan, fast-track and rare pediatric disease designations. We will continue to work closely with the FDA in the coming months with the goal of making our plasminogen replacement therapy Ryplazim™ available to patients as soon as possible”.
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 for a rare pediatric disease priority review voucher (PRV). The grant of the priority review was another important pre-requisite for Prometic’s eligibility for a PRV.
Prometic has previously reported data from this pivotal Phase 2/3 trial, which showed that RyplazimTM treatment consistently replaced and maintained the plasminogen concentration at an appropriate level and that it resolved all lesions in all patients treated.
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.'
Jim |
