Date: 2015-06-01
Type of information: Presentation of results at a congress
phase: 2
Announcement: presentation of results at the 9th C1- Inhibitor Deficiency Workshop in Budapest
Company: Pharming (The Netherlands)
Product: Ruconest® (Rhucin® in non-European territories - conestat alfa)
Action
mechanism: enzyme inhibitor. Ruconest® is a recombinant version of the human protein C1 inhibitor (C1INH). It is produced through Pharming’s proprietary technology in milk of transgenic rabbits.
Disease: acute attacks of angioedema in pediatric patients with Hereditary Angioedema (HAE)
Therapeutic area: Genetic diseases - Rare diseases
Country:
Trial
details: The protocol for the pediatric study has been agreed with the Pediatric Committee of the European Medicines Agency (EMA). The study will assess the pharmacokinetic, safety and efficacy profiles of Ruconest® at a dose of 50 U/kg in pediatric HAE patients in support of a pediatric indication for treatment of HAE attacks. Pharming expects to enroll approximately 20 pediatric patients by the end of the year, aged from 2 to 13 years.
Latest
news: * On June 1, 2015. Pharming announced the presentation of new results, supporting its EMA and FDA approved Hereditary Angioedema (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. * On February 10, 2012, Pharming announced that the company has started an open-label Phase II clinical study evaluating its recombinant human C1 inhibitor Ruconest® (Rhucin® in non-European territories - conestat alfa) for the treatment of acute attacks of angioedema in pediatric patients with Hereditary Angioedema (HAE).
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.
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. The titles of the presentations are: Immunogenicity of recombinant human C1INH in Subjects with Allergies to Cow’s Milk or Rabbits – Oral Presentation by Dr. Mignon van den Elzen. (Division of Internal Medicine and Dermatology, University Medical Center Utrecht, the Netherlands) and Safety and Efficacy of Recombinant Human C1 inhibitor for the Treatment of HAE Attacks in Pediatric Patients– Oral Presentation by Dr. Avner Reshef. (Sheba Medical Center, University of Tel Aviv, Israel)
Full session details and data abstracts for the 2015 Bi-Annual Meeting can be found on the C1 Inhibitor Deficiency Workshop website at http://haenet2015.hu/.(abstracts)
