antomaat schreef op 29 november 2013 22:32:
Pharming Group NV Key Developments
Santarus and Pharming Group Announce Encouraging Data from Phase III HAE Study
Nov 28 13
Santarus, Inc. and Pharming Group NV have announced new data from an open-label extension of the pivotal Phase III clinical study with RUCONEST, or recombinant human C1 esterase inhibitor, or rhC1INH. RUCONEST was administered for the treatment of 224 repeat acute angioedema attacks in 44 patients with hereditary angioedema (HAE) following initial treatment in the pivotal randomized controlled clinical study. RUCONEST is an investigational drug in the US and has been granted orphan drug designation by the FDA for the treatment of acute attacks of HAE. The median time in minutes (95% confidence interval [CI]) 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 (48, 90) to 134.0 (75, 150) and across all attacks was a median of 75.0 (69, 89). The median (95% CI) time in minutes to minimal symptoms (first three attacks per patient) as measured by a TEQ ranged from 243 (76, 1440) to 304 (150, 719) and for all assessed attacks was 303 (211, 367). Only one dose of RUCONEST was administered for 96% of the 224 attacks. 12 of 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 US 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 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 US that has been granted orphan drug designation by the FDA