Pharming Announces Interim Results From the Ongoing Phase II Pediatric Clinical Trial of RUCONEST
LEIDEN, The Netherlands, June 1, 2015 /PRNewswire/ --
- Presentations at the 9th C1-Inhibitor Deficiency Workshop on supportive (interim) results of the use of RUCONEST for the treatment of acute HAE attacks in children under 13 years, alongside data further defining safety and efficacy of RUCONEST -
Pharming Group NV ("Pharming" or "the Company") (Euronext: PHARM) announced today 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.
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."
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)
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/.
About HAE
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 diarrhea caused by swelling of the intestinal wall.
Edema of the throat, nose or tongue is particularly dangerous and potentially life-threatening and can lead to obstruction of the airway passages. 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.
About RUCONEST®
RUCONEST® (C1 Esterase Inhibitor [Recombinant]/ conestat alfa) 50 IU/kg is an injectable medicine that is used to treat acute angioedema attacks in adult and adolescent patients with hereditary angioedema (HAE). HAE is caused by a deficiency of the C1 esterase inhibitor protein, which is present in blood and helps control inflammation (swelling) and parts of the immune system. A shortage of C1 esterase inhibitor can lead to repeated attacks of swelling, pain in the abdomen, difficulty breathing and other symptoms. RUCONEST® contains C1 esterase inhibitor at 50 IU/kg.
When administered at the onset of HAE attack symptoms at the recommended dose, RUCONEST works to return a patient's C1-INH levels to normal range and quickly begins to relieve the symptoms of an HAE attack with a low recurrence of symptoms. RUCONEST is the first and only plasma-free, recombinant C1-INH approval from the U.S. Food and Drug Administration (FDA) and was approved in July 2014 and by the European Medicines Agency (EMA) in October 2010.
About Pharming Group NV
Pharming Group NV is developing innovative products for the treatment of unmet medical needs. RUCONEST® (conestat alfa) is a recombinant human C1 esterase inhibitor approved for the treatment of angioedema attacks in patients with HAE in the USA, Israel all 27 EU countries plus Norway, Iceland, and Liechtenstein.
RUCONEST is commercialized by Pharming in Austria, Germany and The Netherlands. RUCONEST is distributed by Swedish Orphan Biovitrum AB (publ) (SS: SOBI) in the other EU countries, and in Azerbaijan, Belarus, Georgia, Iceland, Kazakhstan, Liechtenstein, Norway, Russia, Serbia, and Ukraine.
RUCONEST is partnered with Salix Pharmaceuticals, Ltd. ("Salix") in North America. Valeant Pharmaceuticals International, Inc. (NYSE: VRX/TSX: VRX) completed its acquisition of Salix Pharmaceuticals, Ltd. on April 1, 2015.
RUCONEST is also being investigated in a randomized Phase II clinical trial for prophylaxis of HAE, in a phase II clinical trial for the treatment of HAE in young children (2-13 years of age) and evaluated for various additional follow-on indications.
Pharming has a unique GMP compliant, validated platform for the production of recombinant human proteins that has proven capable of producing industrial volumes of high quality recombinant human protein in a more economical way compared to current cell-based technologies. Leads for Enzyme Replacement Therapy (ERT) in Pompe, Fabry's and Gaucher's diseases are under early evaluation. The platform is partnered with Shanghai Institute of Pharmaceutical Industry (SIPI), a Sinopharm Company, for joint global development of new products. Pre-clinical development and manufacturing will take place at SIPI and are funded by SIPI. Pharming and SIPI initially plan to utilise this platform for the development of recombinant human Factor VIII for the treatment of Haemophilia A.
For more information, please visit http://www.pharming.com
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