Liminal BioSciences Inc. (NASDAQ: LMNL) vaulted early Monday on news that the U.S. Food & Drug Administration (FDA) has approved Ryplazim ® (plasminogen, human-tvmh) 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.
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.
Said CEO Bruce Pritchard, "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."
LMNL shares climbed $1.75, or 43.4%, to $5.78.
