The weekly peer-reviewed general medical journal The Lancet has published data from a Phase II, double-blind, placebo-controlled, randomized clinical trial (NCT02247739) evaluating the efficacy and safety of RUCONEST (C1 esterase inhibitor [recombinant]) for the prevention of HAE attacks.
As previously reported, in a study with 32 patients RUCONEST® 50 IU/kg (max 4200 IU) demonstrated a statistically significant and clinically relevant reduction in attack frequency for both the twice-weekly and once-weekly treatment regimens when compared to placebo and was generally safe and well-tolerated in the study.
Lead-author and co-principal investigator, Marc Riedl, MD, Professor of Medicine and Clinical Director at the US HAEA Angioedema Center at The University of California San Diego, commented: “Patients with frequent HAE attacks, such as those enrolled in this study, are severely affected and have limited safe and effective choices to control their disease. The findings from this positive study emphasize the unique attributes of recombinant C1INH, and support the potential prophylactic benefits of the medication.”
Dr. Bruno Giannetti, MD, Chief Operations Officer of Pharming Group N.V., added: “We are pleased to see these important results published in a leading peer-reviewed journal. We wish to thank the patients and researchers involved in this study, and we look forward to continuing our work with the HAE community to improve treatment options.”
RUCONEST® is a recombinant C1 esterase inhibitor (C1-INH) indicated for the treatment of acute attacks in adult and adolescent patients with HAE. The product was granted Food and Drug Administration approval in this indication on 17 July 2014. RUCONEST® addresses the cause of HAE attacks by increasing C1-INH in the plasma to normal levels and by stopping the production of kallikrein, an enzyme that activates bradykinin and causes blood vessels to leak.
The complete publication can be found at: http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(17)31963-3/fulltext?elsca1=tlxpr