Ruconest Effective in HAE study

Santarus and Pharming Group will present new data from an open-label extension of the Phase III trial of Ruconest (recombinant human C1 esterase inhibitor, or rhC1INH) at the 2013 American College of Allergy, Asthma & Immunology Annual Scientific Meeting.

Ruconest was administered for the treatment of 224 repeat acute angioedema attacks in 44 patients with hereditary angioedema (HAE) following initial treatment in the randomized controlled Phase III study.

The median time in minutes to onset of symptom relief following treatment as measured by patient responses to a Treatment Effect Questionnaire (TEQ) for the first five repeat attacks ranged from 62.5 to 134.0 and across all attacks was a median of 75.0. The median time in minutes to minimal symptoms (first three attacks per patient) as measured by a TEQ ranged from 243 to 304 and for all assessed attacks was 303.

Only one dose of Ruconest was administered for 96% of the 224 attacks. Twelve of the 44 (27%) patients experienced at least one treatment emergent adverse event (TEAE) within 72 hours of completion of Ruconest infusion. TEAEs occurring in 5% of patients were nasopharyngitis, cough, fibrin D-dimer increase, and headache. The percentage of patients experiencing TEAEs did not increase with Ruconest treatments for repeat attacks. There were no discontinuations due to adverse events, no thrombotic or anaphylactic events, and no neutralizing anti-C1INH antibodies observed with repeat Ruconest treatment.

Santarus and Pharming are seeking U.S. marketing approval of Ruconest for the treatment of acute angioedema attacks in patients with HAE. The Biologics License Application (BLA) filing for Ruconest is under review by the U.S. Food and Drug Administration (FDA) with a response expected by April 16, 2014. Ruconest is approved in Europe for the treatment of acute angioedema attacks in patients with HAE and is an investigational drug in the U.S. that has been granted orphan drug designation by the FDA.