Santarus and Pharming Group have announced positive results from their Phase III study of Ruconest conducted in patients with Hereditary Angioedema (HAE).
The study, which evaluated the safety and efficacy of Ruconest (recombinant human C1 esterase inhibitor) 50 U/kg, has met the primary endpoint of time to beginning of symptom relief.
Santarus research and development executive vice president Dr Wendell Wierenga said; "These positive results are consistent with the efficacy data previously reported from two smaller randomised, 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."
The intent-to-treat population (n=75) between Ruconest and placebo (p=0.031, log-rank test) demonstrated a statistically significant difference in the time to beginning of symptom relief.
A median time of 90 minutes was observed in Ruconest patients for the beginning of symptom relief and 152 minutes for placebo patients.
The time to beginning of symptom relief was defined as the time from the beginning of infusion of study medication (Ruconest or placebo) until the beginning of a persistent beneficial effect, based on the patient's responses to a treatment effect questionnaire for the primary attack location.
Ruconest was generally well tolerated, although within 72 hours of the completion of infusion of study medication, four Ruconest patients (22%) experienced six adverse events in the form of lipoma, back pain, sneezing, procedural headache, skin burning sensation and an increase in fibrin D-dimer.
Pharming chief operations officer Dr Bruno Giannetti said the company is looking forward to working with Santarus to prepare and submit Ruconest's Biologics License Application to the FDA in the first half of 2013.
"We anticipate that additional data from this Phase III study will be presented at an appropriate medical meeting in 2013," Giannetti added.