FDA Grants Orphan-Drug Designation to ProMetic's Plasma-Derived Plasminogen Drug
ProMetic Life Sciences Inc. announced today that an orphan drug designation status has been granted for its plasma purified human plasminogen drug to ProMetic BioTherapeutics, Inc., its US based subsidiary, by the FDA. The orphan drug designation is for the treatment of hypoplasminogenemia, or type I plasminogen deficiency ("T1PD").
ProMetic is currently developing a systemic formulation for the treatment of T1PD in partnership with Hematech Biotherapeutics Inc. ("Hematech") and has already successfully scaled up the manufacturing process.
T1PD is a rare autosomal recessive disorder that leads to severe clinical manifestations, primarily associated with fibrous depositions on mucous membranes throughout the body. It is a multisystem disease that affects the eyes, ears, sinuses, tracheobronchial tree, genitourinary tract and gums. There is no effective replacement therapy currently available for this disease.
"The conditions resulting from the deficiency of plasminogen require that several medical specialties combine their efforts to provide care to these patients", stated Dr Tom Chen, Senior Vice President, Product & Asian Market Development. "We are very proud to bring this tangible therapeutic solution enabled by our proprietary manufacturing platform and our strategic partner, Hematech Biotherapeutics", added Dr Chen.
"Given the multitude of organs and tissues that are affected by such type of disorders, it is critical to be able to provide a safe and reliable source of plasminogen for these patients in need", stated Pierre Laurin, CEO of ProMetic. "The scale-up of our proven manufacturing platform at our Laval, Quebec facility will enable the global supply of plasminogen as well as several other therapeutics targeting serious unmet medical conditions".
Orphan Drug Designation is granted to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven year period of U.S. marketing exclusivity upon marketing approval for the designated indication, as well as with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and the waiver of prescription drug user fees.