Liminal BioSciences Announces Resubmission of Biologics License Application to FDA for Ryplazim

Liminal BioSciences announced that the company, through its U.S. subsidiary Prometic Biotherapeutics, has filed a resubmission of the biologics license application (BLA) for Ryplazim (plasminogen) (Ryplazim) with the FDA for the treatment of congenital plasminogen deficiency (C-PLGD).

“The resubmission of this BLA represents a significant milestone for Liminal BioSciences and we believe it has meaningful potential for patients and families affected by congenital plasminogen deficiency,” Kenneth Galbraith, Chief Executive Officer of Liminal BioSciences, said in a company news release. “We look forward to continuing to work with the FDA toward our goal of achieving regulatory approval and making Ryplazim available to patients with this congenital rare disease in the United States.”

C-PLGD is an ultra-rare disorder that results in inflamed fibrinous growths on mucous membranes throughout the body, most often affecting tissue around the eye but also in the mouth, nasal cavity, gastrointestinal, bronchial and genitourinary tracts. In some cases, lesions can lead to blindness or be life-threatening. There are currently no FDA approved treatments available.

“C-PLGD is a rare multisystem disorder that can have a profound effect on a patient’s health and quality of life. With no approved treatment available, C-PLGD is an area of significant unmet need throughout the world,” Dr. Amy Shapiro, Chief Executive Officer and Co-medical Director at the Indiana Hemophilia & Thrombosis Center, and principal investigator in the pivotal clinical trial supporting the BLA resubmission, said in the news release.

In a pivotal phase 2/3 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.