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Positive Phase III Results for Ruconest in Hereditary Angioedema
Treated patients experienced symptom relief sooner compared with placebo group (Nov. 7)
A pivotal phase III clinical study of the investigational drug Ruconest (recombinant human C1 esterase inhibitor; Santarus, Inc.) 50 U/kg for the treatment of acute attacks of angioedema in patients with hereditary angioedema (HAE) has met its primary endpoint of the time to the beginning of symptom relief.
HAE is a genetic disorder in which patients are deficient in or lack a functional plasma protein C1 esterase inhibitor, resulting in unpredictable and debilitating episodes of intense swelling. The swelling may occur in one or more anatomical areas, including the extremities, face, trunk, genitals, abdomen, and upper airway. The frequency and severity of HAE attacks vary and are most serious when they involve laryngeal edema, which can close the upper airway and cause death by asphyxiation. According to the U.S. Hereditary Angioedema Association, epidemiological estimates for HAE range from one in 10,000 to one in 50,000 individuals.
In the phase III study, a statistically significant difference in the time to the beginning of symptom relief was observed in the intent-to-treat population between Ruconest (n = 44) and placebo (n = 31); the median time to the beginning of symptom relief was 90 minutes for the Ruconest-treated patients and 152 minutes for those given placebo (P = 0.031). The time to the beginning of symptom relief was defined as the time from the beginning of infusion of the study medication (Ruconest or placebo) until the beginning of a persistent beneficial effect, based on the patient's responses to a Treatment Effect Questionnaire for the primary attack location.
Within 72 hours after completion of the infusion, four Ruconest-treated patients (7%) experienced six adverse events: sneezing, procedural headache, back pain, burning sensation of the skin, increased fibrin D-dimer, and lipoma. Within the 72-hour post-infusion period, four placebo-treated patients (22%) experienced four adverse events: sinus congestion, vasomotor rhinitis, diarrhea, and dyspepsia. Thromboembolic events, anaphylaxis, or neutralizing antibodies to the C1 inhibitor were not observed in any patient.
Ruconest is produced using milk from transgenic rabbits. The drug is approved in Europe for the treatment of acute angioedema attacks in patients with HAE.
Ruconest has been granted orphan drug designation by the FDA both for the treatment of acute attacks of HAE and for prophylactic treatment of HAE.
Source: Santarus, Inc.; November 7, 2012.