Pharming Group N.V. announces positive data from a clinical trial with the use of Ruconest [Recombinant Human C1 Esterase Inhibitor/ conestat alfa] for the treatment of HAE attacks in children.
The open-label, single arm, Phase II clinical trial was designed in agreement with the European Medicines Agency (EMA) as part of a Paediatric Investigation Plan (PIP) to assess the pharmacokinetic, safety and efficacy profiles of Ruconest at a dose of 50 IU/kg in paediatric HAE patients ages 2-13 years in support of a paediatric indication for treatment of HAE attacks.
A total of 20 children with HAE were treated for 73 HAE attacks at a dose of 50 IU/kg (up to a maximum of 4200 IU). The study reported clinically meaningful relief of symptoms assessed using a visual analogue scale (VAS) completed by the patient (assisted by their parent). The median time to onset of relief was 60 minutes (95% confidence interval: 60-63), and the median time to minimal symptoms was 122 minutes (95% confidence interval: 120-126). Only 3/73 (4%) attacks were treated with a second dose of Ruconest.
Ruconest was generally safe and well-tolerated in the study. No patients withdrew from the study due to adverse events. There were no related serious adverse events, hypersensitivity reactions, or neutralizing antibodies detected.
The detailed efficacy and safety results will be further analyzed and submitted for presentation at an upcoming major medical meeting.
Prof. Bruno Giannetti, Pharming’s Chief Operations Officer, commented:
“The results of this study provide important safety and efficacy data on the use of Ruconest in children and show good clinical concordance with results from adolescents and adults with HAE. Children with HAE have limited therapeutic options, and we will work expeditiously with regulatory authorities to expand choices for these patients.”