Biotech company Pharming Group NV announced that, as part of the activities for the regulatory submission files, it has now also completed the final analysis of its Rhucin randomised placebo-controlled studies. The results confirm the positive and consistent efficacy and safety of Rhucin in the treatment of acute angioedema attacks in patients with Hereditary Angioedema (HAE).
Pharming conducted two independent, randomized, double blind, placebo controlled clinical studies in Europe and in North-America (US and Canada). In total, 70 attacks were randomized to one of two doses of Rhucin (100 U/kg, 50 U/kg body weight) or placebo control treatment. Of these 70 patients, 32 were treated in the European study and 38 patients in the North-American study. The final statistical analyses have now been undertaken in line with the latest EU and US regulatory guidance. Both studies independently demonstrated statistically significant and clinically relevant results on primary and secondary endpoints, thus demonstrating the efficacy of Rhucin for the treatment of acute attacks of Hereditary Angioedema.
In the EU study, time to onset of relief (median), the primary endpoint, was 62 minutes for Rhucin 100 U/kg compared to 508 minutes for control treatment. In the North America study, time to onset of relief (median) was 68 and 122 minutes respectively for Rhucin 100 and 50 U/kg compared to 258 minutes for control treatment. In the EU study, time to minimal symptoms (median), the secondary endpoint, was 480 minutes for Rhucin 100 U/kg compared to 1440 for control treatment. In the North America study, time to minimal symptoms (median) was 245 and 247 minutes respectively for Rhucin 100 and 50 U/kg compared to 1101 for control treatment. None of the Rhucin treated patients experienced a relapse of symptoms in the same attack.
As to safety, no clinically relevant concerns were reported in either study. There were no drug-related serious adverse events, no allergic reactions, and, no immuno-safety concerns due to antibody responses to Rhucin or rabbit milk proteins. Rhucin infusions were well tolerated, without reports of pain or irritation at the injection site. Together with the evaluation of the open-label studies and other studies reported previously, the Rhucin safety database now includes over 300 treatments of acute attacks.
"The results from these studies demonstrate Rhucin's excellent efficacy and safety profile in treating acute attacks of HAE," said Marco Cicardi, professor of Internal Medicine at the University of Milan, Italy and Principal Investigator of the European trial. "Having a recombinant human C1 inhibitor protein, such as Rhucin, as a treatment option for HAE is a notable advance in the field that will be important to both patients and physicians."
"Rhucin has shown to safely provide rapid and sustained relief of symptoms in HAE patients having acute attacks. We believe that our current clinical database adequately supports the efficacy and safety of Rhucin, in single and repeated use, in acute attacks at all anatomical locations, including laryngeal attacks," said Bruno Giannetti, chief operations officer of Pharming. "With the European regulatory agency recently agreeing on the paediatric investigation plan for Rhucin in HAE and the recently reported immuno-safety data, we are on schedule to submit our European Union Marketing Authorization Application for Rhucin in September 2009."
The open-label extension studies of Rhucin are ongoing in HAE patients for the treatment of acute attacks. Over 380 acute angioedema attacks of all types in over 130 patients have now been treated including many potentially life-threatening laryngeal attacks. More than 25 HAE patients have been treated at least 4 times and one HAE patient has been treated 25 times.
Pharming Group NV is developing innovative products for the treatment of genetic disorders, ageing diseases, specialty products for surgical indications, intermediates for various applications and nutritional products.