Brussels-based UCB SA said its experimental drug rozanolixizumab helped patients with primary immune thrombocytopeni achieve clinically relevant improvements in platelet count and reduced immunoglobin G levels in a mid-stage trial called TP0001.
Primary immune thrombocytopeni is an autoimmune bleeding disorder in which the blood does not clot due to a low number of platelets. Symptoms mainly include spontaneous bruising, bleeding and fatigue.
Rozanolixizumab, which was found to be safe and well-tolerated in patients, was administered in multiple doses, and subjects were monitored for an eight-week observation period after completing treatment. The company found improvements in platelet count across all dose groups and decreases in immunoglobin G, which is responsible for the removal and destruction of new or existing platelets in the body.
UCB presented the data at the 2019 American Society of Hematology meeting. The company's medicine was granted the orphan designation by the U.S. Food and Drug Administration in 2018 and the European Commission earlier this year.