The US FDA approved Ryplazim of Prometic Biotherapeutics Inc, a subsidiary of Liminal BioSciences Inc (NASDAQ: LMNL), to treat patients suffering from hypoplasminogenia.
Ryplazim is the first approved drug in the market to treat this rare genetic disorder. Liminal assessed Ryplazim efficacy in adult patients and pediatric patients with hypoplasminogenia in an open-label and single-arm clinical study.
Enrolls 15 patients
The company enrolled 15 patients with baseline plasminogen activity level in a window of less than 5% and 45% for this clinical trial. It administered a 6.6 mg/kg dose in patients every two to four days over 48 weeks to accomplish 10% individual trough plasminogen activity improvement above the baseline and cure the disease clinical manifestations.
The drug is well tolerated in the subjects in the clinical trial. It established the efficacy based on clinical success at the 48th week.
At least 50% of patients with measurable non-visible or visible lesions achieved a 50% enhancement in lesion size/ number in this clinical trial.
Also, the patients with any lesion demonstrated 50% improvement in the size or number of their lesions. Liminal’s CEO, Bruce Pritchard, said the FDA approval for Ryplazim is the first milestone for its patients, physicians, caregivers, and the company.
Ryplazim is available for patients in the US
The company is excited that its Ryplazim will be available for US patients with congenital plasminogen deficiency. Liminal could improve its non-dilutive cash to advance its small molecule R&D strategy following the Rare Pediatric Disease PRV (Priority Review Voucher).
Liminal’s President, Patrick Sartore, said its innovative drug – Ryplazim, could now offer an effective cure for families and patients living with plasminogen type 1 deficiency and helps to manage their symptoms.
He further said that FDA approval for Ryplazim is an important milestone in providing much-needed treatment for patients suffering from this rare genetic disorder.
Patients suffering from plasminogen deficiency experience reduced levels of plasma plasminogen.
The liver synthesizes this naturally occurring protein – Plasminogen aka plasmin and circulates it in the blood. Plasmin engages in the lysis of the clot and clears extravasated fibrin.
The company previously received Rare Pediatric Disease and Orphan drug designations from the US FDA to treat congenital plasminogen deficiency.