Liminal BioSciences Provides Update on Progress on BLA for Ryplazim (Plasminogen)

Liminal BioSciences announced that the FDA acknowledged receipt of the resubmission of the Biologic License Application (BLA) for Ryplazim (plasminogen) for the treatment of clinical signs and symptoms associated with congenital plasminogen deficiency (C-PLGD) in pediatrics and adults.

The submission was made by Liminal BioSciences’ subsidiary Prometic Biotherapeutics. The FDA confirmed that the resubmission is a complete, Class 2 response and has provided a Prescription Drug User Fee Act (PDUFA) target action date of March 5, 2021.

“We look forward to continuing to work closely with FDA during the BLA review process for Ryplazim as we move one step closer to potentially bringing the first FDA-approved treatment to patients with this devastating disease,” Kenneth Galbraith, Chief Executive Officer of Liminal BioSciences, said in a company news release.

The BLA resubmission for Ryplazim is supported by data from a phase 2/3 clinical study which evaluated 15 patients, both pediatric and adults, with congenital plasminogen deficiency over 48 weeks of therapy with Ryplazim. In the phase 2/3 clinical study, 100% of the patients enrolled met both primary efficacy endpoints of the clinical study. 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.

“In parallel to the FDA review process, we will build our commercial readiness for a potential approval and potential commercial launch in the US in 2021,” stated Patrick Sartore, Chief Operating Officer – Plasma-Derived Therapeutics. “We are continuing our work to build awareness of congenital plasminogen deficiency with patient advocacy groups, physicians and payors to enable patient access in the US if Ryplazim is approved by the FDA. In addition, we are exploring development and regulatory pathways in countries outside the US to seek marketing approval for Ryplazim with potential third-party collaboration partners.”

Under the FDA’s rare pediatric disease designation program, the FDA may grant a priority review voucher (PRV) to a sponsor who receives a product approval for a “rare pediatric disease” which is defined as a serious life-threatening disease in which the serious or life-threatening manifestations primarily affects fewer than 200,000 people in the U.S. Subject to FDA approval of Ryplazim for the treatment of C-PLGD, the company may be eligible to receive one priority review voucher, which then could be redeemed to receive priority review for any subsequent marketing application, or sold or transferred to other companies for their programs.