Pharming Group NV (NYSE Euronext: PHARM) and Santarus, Inc. (NASDAQ: SNTS) announced that new data from an open-label extension of the pivotal Phase III clinical study with RUCONEST® (recombinant human C1 esterase inhibitor, or rhC1INH) will be featured in a poster presentation on November 9 & 10, 2013 at the 2013 American College of Allergy, Asthma & Immunology Annual Scientific Meeting at the Convention Center in Baltimore, Maryland. The poster is titled, Efficacy and Safety of Recombinant Human C1 Esterase Inhibitor for Acute Attacks of Hereditary Angioedema: An Open-Label Study.
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 U.S. and has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of acute attacks of HAE.
"The results from this clinical trial are consistent with findings from previous studies supporting that RUCONEST reduces time to symptom relief when used for repeat HAE attacks," said H. Henry Li, M.D., Ph.D., Director of Chevy Chase Clinical Research, Institute for Asthma and Allergy, Chevy Chase, Maryland. "In addition, it is important clinically to evaluate the safety findings from these multiple exposures."
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).
Exposure and Safety Information
- 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 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.
About RUCONEST and Hereditary Angioedema
RUCONEST (INN conestat alfa) is a recombinant version of the human protein C1 esterase inhibitor, and is produced with Pharming's proprietary transgenic technology. RUCONEST is approved in Europe for the treatment of acute angioedema attacks in patients with HAE, a genetic disorder in which the patient is deficient in or lacks 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.