Pharming Group NV presented new results, supporting its EMA and FDA approved HAE therapy RUCONEST®, at the 9th C1- Inhibitor Deficiency Workshop that took place in Budapest, Hungary, 28-31 May 2015.

Several abstracts, including two oral presentations, were presented, which demonstrate Pharming’s ongoing commitment to advance innovative science in HAE, with the goal of addressing significant clinical needs and improving patient care.

The presentations featured interim data from the ongoing pediatric clinical trial and results from a clinical immunology study, which support the safety and efficacy of RUCONEST® [Recombinant Human C1 Esterase Inhibitor/ conestat alfa].

The ongoing pediatric study is an open label Phase II clinical trial assessing safety, immunogenicity and efficacy in children 2-13 years of age with C1INH deficiency. Eight children were treated on demand for 28 HAE attacks at 50 IU/kg body weight (up to a maximum of 4200 IU). Efficacy endpoints were time to onset of relief and to minimal symptoms, assessed by the patient (assisted by their parent), using a visual analogue scale (VAS) and by physicians using an Investigator Score. Median time to beginning of relief was 60 minutes as determined by the patients and the investigators. Using the VAS, 93% of patients had onset of relief within 2 hours. No related serious adverse events, including hypersensitivity reactions, were reported.

“We believe the additional body of pediatric clinical data in children under 13 years of age are in line with the excellent data in adolescents (13-18 years of age) and adult HAE patients treated with RUCONEST® for acute attacks,” said Dr. Avner Reshef MD, Sheba Medical Center, University of Tel Aviv, Israel. “This clinical research will further enhance physicians’ trust in recombinant human C1INH therapies”.

The second study investigated the immunogenicity of host (rabbit) related impurities (<0.002%) in 26 subjects with allergies to cow’s milk and/or rabbit dander. Subjects were challenged with increasing doses of rhC1INH by skin prick, followed by intra-cutaneous injections, and finally sub-cutaneous challenge. No subjects (with pre-existing rabbit allergy or cow’s milk allergy) had any confirmed clinical and laboratory evidence of hypersensitivity to rhC1INH.

“These data add to our clinical database and post-marketing experience that demonstrate a very low risk of allergy with rhC1INH exposure,” said Prof. Dr. Giannetti, MD PhD, Pharming’s Chief Operating Officer.

“I am very pleased with the additional RUCONEST clinical efficacy and safety data” said Sijmen de Vries, MD, Pharming’s CEO: “These achievements are a major step forward to supporting a “Recombinant for all” concept, which is already increasingly implemented in other rare diseases, such as haemophilia, driven by the continued concerns of emerging pathogens in blood-derived products.”

(Source: Pharming Group)