Positive results from Phase III trial of Ruconest (Santarus) in patients with Hereditary Angioedema

A pivotal Phase III clinical study to evaluate the safety and efficacy of the investigational drug Ruconest (recombinant human C1 esterase inhibitor), from Santarus, for the treatment of acute attacks of angioedema in patients with Hereditary Angioedema (HAE) met the primary endpoint of time to beginning of symptom relief. 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).

The drug was generally well tolerated and the frequency of patients experiencing at least one treatment emergent adverse event in the Ruconest treated group was less than in the placebo group. Within 72 hours of the completion of infusion of study medication, four Ruconest patients (7%) experienced six adverse events: sneezing, procedural headache, back pain, skin burning sensation, an increase in fibrin D-dimer and lipoma. Santarus will submit the Biologics License Application (BLA) for Ruconest to the FDA in the first half of 2013 and additional data from this Phase III study will be presented at a medical meeting in 2013.