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June 4, 2021Ryplazim Approved to Treat Rare Genetic Disorder
June 4, 2021 – The U.S. FDA has approved Ryplazim® (plasminogen, human-tvmh), manufactured by Liminal BioSciences, to treat plasminogen deficiency type 1 (hypoplasminogenemia). Ryplazim is the first FDA-approved treatment for the disease.
Hypoplasminogenemia is a rare, potentially fatal genetic disorder that causes a lack of plasminogen, an enzyme the body needs to break down fibrin (a protein formed when blood clots). This leads to thick growths (lesions) on the body’s mucous membranes that can interfere with normal organ function. The growths can affect vision, the digestive tract, the respiratory system, and other parts of the body with varying severity. Ryplazim treats the disease by replacing absent plasminogen.
In a clinical trial, patients who had lesions at baseline had at least a 50% improvement in lesion number/size when treated with Ryplazim. New or recurrent lesions were absent in all patients (regardless of whether they had lesions at baseline) through 48 weeks of Ryplazim treatment during the trial. Recommended dosing is 6.6mg/kg of the patient’s body weight administered via intravenous infusion every two to four days.
Launch and pricing information have not yet been announced.