Santarus and Pharming Group NV announced that their pivotal Phase 3 clinical study to evaluate the safety and efficacy of the investigational drug Ruconest (recombinant human C1 esterase inhibitor) for the treatment of acute attacks of angioedema in patients with Hereditary Angioedema (HAE) met its primary endpoint. Ruconest (INN conestat alfa) is a recombinant version of the human protein C1 esterase inhibitor.
Study 1310 was an international, multicenter, randomized, placebo-controlled study comparing a single IV infusion of 50U/kg of Ruconest to a saline control with a primary endpoint of time to beginning of symptom relief. The time to beginning of symptom relief was defined as the time lapsed from the beginning of the infusion of study medication to the beginning of a persistent beneficial effect based on the patient’s responses to a Treatment Effect Questionnaire for the primary attack location. A 90 day follow-up period was required for each patient enrolled, or until such time that the patient required open-label treatment for a subsequent attack during the 90 day period. The study enrolled a total of 75 patients who were randomized 3:2 to receive either Ruconest or saline.
A statistically significant difference in the time to beginning of symptom relief was observed in the intent-to-treat population (n=75) between Ruconest and placebo (P=0.031, log-rank test); the median time to beginning of symptom relief was 90 minutes for Ruconest patients (n=44) and 152 minutes for placebo patients (n=31).