Liminal BioSciences announces FDA approval for its BLA of Ryplazim to treat patients with plasminogen deficiency type 1.

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 less than 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. 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.