LAVAL, CANADA, and CAMBRIDGE, ENGLAND – Liminal BioSciences Inc. (Nasdaq: LMNL) (“Liminal BioSciences” or the “Company”) announced today that the U.S. Food & Drug Administration (FDA) has approved Ryplazim® (plasminogen, human-tvmh) (“Ryplazim®”) for the treatment of patients with plasminogen deficiency type 1 (hypoplasminogenia) through its subsidiary, Prometic Biotherapeutics Inc., holder of the biological license application (“BLA”) for Ryplazim®. With this approval, Ryplazim® becomes the first FDA approved therapy for this rare genetic disorder.
The efficacy of Ryplazim® in pediatric and adult patients with plasminogen deficiency type 1 was evaluated in a single-arm, open-label clinical trial. A total of 15 patients who had a baseline plasminogen activity level between <5% and 45% of normal were enrolled. All patients received Ryplazim® at a dose of 6.6 mg/kg administered every two to four days for 48 weeks to achieve at least an increase of individual trough plasminogen activity by an absolute 10% above baseline and to treat the clinical manifestations of the disease. Ryplazim® was well tolerated in the clinical study (See Important Safety Information).
Efficacy was established on the basis of overall rate of clinical success at 48 weeks defined as 50% of patients with visible or other measurable non-visible lesions achieving at least 50% improvement in lesion number/size, or functionality impact from baseline. All patients with any lesion at baseline showed at least 50% improvement in the number or size of their lesions.
“Receiving our first drug approval is a major milestone for Liminal and for the patients, caregivers, and physicians who have been with us every step of the way in this important research effort. We are very pleased that Ryplazim® will be available to US patients suffering from congenital plasminogen deficiency,” said Bruce Pritchard, the CEO of Liminal BioSciences. “The receipt of a Rare Pediatric Disease Priority Review Voucher (PRV) also has the potential to provide Liminal with non-dilutive cash to support our ongoing efforts to advance and expand our small molecule R&D strategy”.
“This approval gives patients and families who live with plasminogen type 1 deficiency a new option to try to manage symptoms,” stated Mr. Patrick Sartore, President of Liminal BioSciences. “This FDA approval marks an important turning point, providing a first and much-needed therapy for patients with this rare genetic disease.”
In all patients with plasminogen deficiency, plasma plasminogen levels are markedly reduced. Plasminogen is a naturally occurring protein that is synthesized by the liver and circulates in the blood. Activated plasminogen, known as plasmin, is an enzymatic component of the fibrinolytic system and the main enzyme involved in the lysis of clots and clearance of extravasated fibrin.
As part of the sale of Liminal BioSciences’ plasma collection centers operated in Winnipeg, Manitoba and Amherst, New York in May 2021, Liminal BioSciences entered into an option agreement with Kedrion S.p.A. (Kedrion) pursuant to which Kedrion has the right to acquire the remainder of Liminal BioSciences’ plasma-derived therapeutics’ business, including the Ryplazim® business, for an exercise price of US$5 million and would entitle Liminal BioSciences to receive up to 70% of the net proceeds which may be received from the sale of a PRV.
Ryplazim® was previously granted Orphan Drug and Rare Pediatric Disease Designations by the FDA for the treatment of congenital plasminogen deficiency. With this approval, the FDA issued a PRV to Liminal BioSciences Inc., through its subsidiary Prometic Biotherapeutics Inc., holder of the BLA. The PRV can be redeemed to receive priority review for any subsequent marketing application or sold or transferred to other companies for their programs.