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| |  FDA Approves Ecallantide for Treatment of Acute Attacks of Hereditary Angio-Oedema in Patients 16 Years and Older CAMBRIDGE, Mass -- December 2, 2009 – The US Food and Drug Administration has approved ecallantide (Kalbitor) for the treatment of acute attacks of hereditary angio-oedema (HAE) in patients aged 16 years and older. HAE is a rare, genetic disorder characterised by severe, debilitating and often painful swelling, which can occur in the abdomen, face, hands, feet and airway. Ecallantide, a potent, selective and reversible plasma kallikrein inhibitor, is the first subcutaneous HAE treatment approved in the United States. As part of product approval, Dyax has, together with the FDA, established a Risk Evaluation and Mitigation Strategy (REMS) programme to communicate the risk of anaphylaxis and the importance of distinguishing between a hypersensitivity reaction and HAE attack symptoms. “By specifically affecting a key mediator of the inflammation, pain, and oedema that are characteristic of HAE attacks, [ecallantide] may be advantageous in offering physicians a targeted approach for treating acute attacks,” said professor Jonathan Bernstein, MD, University of Cincinnati College of Medicine, Cincinnati, Ohio. The approval of ecallantide is based on the results of 2 placebo-controlled, phase 3 clinical studies, known as EDEMA3 and EDEMA4. Patients having an HAE attack at any anatomic location, with at least 1 moderate or severe symptom, were treated with ecallantide 30 mg subcutaneous or placebo. Because patients could participate in both trials, a total of 143 unique patients participated. There were 64 patients with abdominal attacks, 55 with peripheral attacks, and 24 with laryngeal attacks. In both trials, the effects of ecallantide were evaluated using the Mean Symptom Complex Severity (MSCS) score and the Treatment Outcome Score (TOS), 2 HAE-specific patient-reported outcome endpoints developed by Dyax. These measures evaluated the severity of attack symptoms at all anatomical locations (MSCS score) and response to therapy (TOS). In the EDEMA4 trial, at 4 hours, patients treated with ecallantide demonstrated a greater decrease from baseline in the mean MSCS than placebo (-0.8 vs. -0.4; P = .010) and a greater mean TOS (53 vs. 8, P = .003). In the EDEMA4 trial, at 24 hours, patients treated with ecallantide also demonstrated a greater decrease from baseline in the mean MSCS than placebo (-1.5 vs. -1.1; P = .04) and a greater mean TOS (89 vs. 55, P = 0.03). The results in the EDEMA3 trial were consistent with the EDEMA4 trial results. Potentially serious hypersensitivity reactions, including anaphylaxis, have occurred in patients treated with ecallantide. In 255 HAE patients treated with intravenous or subcutaneous ecallantide in clinical studies, 10 patients (3.9%) experienced anaphylaxis. For the subgroup of 187 patients treated with subcutaneous ecallantide, 5 patients (2.7%) experienced anaphylaxis. Symptoms associated with these reactions have included chest discomfort, flushing, pharyngeal oedema, pruritus, rhinorrhoea, sneezing, nasal congestion, throat irritation, urticaria, wheezing, and hypotension. These reactions occurred within the first hour after dosing. The most common adverse reactions occurring in >=3% of ecallantide-treated patients and greater than placebo were headache, nausea, diarrhoea, pyrexia, injection site reactions, and nasopharyngitis. HAE attacks, which occur on average more than 20 times yearly, are unpredictable and range in progression and severity. An acute episode may occur in 1 or more anatomical sites, sometimes moving from 1 site to another. “HAE is a highly unpredictable disease because most attacks occur spontaneously with no identifiable trigger. [Ecallantide] will provide patients 16 years of age and older and their physicians with an FDA-approved subcutaneous therapy for treating painful and debilitating HAE acute attacks,” stated Anthony J. Castaldo, President, United States Hereditary Angioedema Association (HAEA), a nonprofit patient advocacy organisation that represents approximately 6,500 HAE patients in the United States. In advance of ecallantide availability and launch, patients and healthcare providers can contact “Kalbitor Access” to receive information and work with programme staff to research patient insurance coverage for ecallantide. Patients and healthcare providers can call 1-888-4KALBITOR (1-888-452-5248) or visit www.KALBITOR.com.
SOURCE: Dyax Corp.
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