Liminal BioSciences announces resubmission of BLA to FDA for Ryplazim to treat congenital plasminogen deficiency.

Liminal BioSciences Inc. announced that the company, through its U.S. subsidiary Prometic Biotherapeutics Inc., has filed a resubmission of the Biologics License Application (BLA) for Ryplazim (plasminogen) with the FDA for the treatment of congenital plasminogen deficiency (C-PLGD). In 2017 the company received a Complete Response Letter (CRL) in response to a BLA submission for Ryplazim. The company believes that the resubmission addresses the deficiencies outlined in the CRL, which were related to certain manufacturing procedures. The company further believes that the amended BLA represents a Class 2 resubmission that would provide for a Prescription Drug User Fee Act (PDUFA) date for FDA review and action within six months from the date of the resubmission. In a pivotal phase II/III clinical trial for the treatment of C-PLGD, the company enrolled 15 patients with C-PLGD, including six pediatric patients, for 48 weeks of therapy with Ryplazim. All of the patients treated with Ryplazim achieved at least the targeted increase from baseline in their individual trough plasminogen activity levels through 12 weeks of therapy. In addition, all patients who had active visible lesions when enrolled in the trial had complete healing of their lesions within 48 weeks of initiating therapy. Adverse events reported in the clinical study were characterized as mild, with no patient deaths, serious adverse events or adverse events that caused study discontinuation reported.