LEIDEN, the Netherlands – September 29, 2017 Pharming Group N.V. ("Pharming" or "the Company") (Euronext Amsterdam: PHARM) announced today positive data from a clinical trial with the use of RUCONEST® [Recombinant Human C1 Esterase Inhibitor/ conestat alfa] for the treatment of hereditary angioedema (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 analysed 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."
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Hereditary Angioedema (HAE) is a rare genetic disorder. It is characterized by spontaneous and recurrent episodes of swelling (edema attacks) of the skin in different parts of the body, as well as in the airways and internal organs. Edema of the skin usually affects the extremities, the face, and the genitals. Patients suffering from this kind of edema often withdraw from their social lives because of the disfiguration, discomfort and pain these symptoms may cause. Almost all HAE patients suffer from bouts of severe abdominal pain, nausea, vomiting and diarrhoea caused by swelling of the intestinal wall.
Edema of the throat, nose or tongue can be particularly dangerous as this can lead to obstruction of the airway passages and be potentially life threatening. Although there is currently no known cure for HAE, it is possible to treat the symptoms associated with edema attacks. HAE affects about 1 in 10,000 to 1 in 50,000 people, worldwide experts believe that a lot of patients are still seeking the right diagnosis: although HAE is (in principle) easy to diagnose, it is frequently identified very late or not discovered at all. The reason HAE is often misdiagnosed is because the symptoms are similar to those of many other common conditions such as allergies or appendicitis by the time it is diagnosed correctly, the patient has often been through a long-lasting ordeal.