RUCONEST was generally well tolerated in this Phase III clinical study 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. Within the 72 hour period four placebo patients (22%) experienced four adverse events: sinus congestion, vasomotor rhinitis, diarrhea and dyspepsia. Thromboembolic events, anaphylaxis, or neutralizing antibodies to C1 inhibitor were not observed in any patient. There was one patient in the RUCONEST group that experienced a serious adverse event (abdominal hernia at Day 79) that was assessed by the investigator as not related to the study drug.
"These positive results are consistent with the efficacy data previously reported from two smaller randomized, controlled clinical studies with RUCONEST in patients with HAE, and we believe the results provide strong support for our proposed dosing regimen of 50 U/kg in treating acute attacks of HAE," said Wendell Wierenga, Ph.D., executive vice president of research and development of Santarus.
"We are very pleased with these pivotal study results and look forward to working with our colleagues at Santarus to prepare and submit the Biologics License Application (BLA) for RUCONEST to the FDA in the first half of 2013," said Bruno Giannetti, M.D., Ph.D., chief operations officer of Pharming. "We anticipate that additional data from this Phase III study will be presented at an appropriate medical meeting in 2013."
Santarus licensed exclusive rights to commercialize RUCONEST in North America for the treatment of acute attacks of HAE as well as other potential future indications from Pharming. Under the terms of the license agreement, a $10 million milestone is now payable to Pharming as a result of the successful achievement of the primary endpoint of the Phase III clinical study. An additional $5 million milestone will be payable to Pharming upon U.S. Food and Drug Administration (FDA) acceptance of the BLA for review.