Date: 2013-11-08
Type of information:
phase: 3
Announcement: presentation of results 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.
Company: Pharming Group (The Netherlands) Santarus (USA)
Product: Ruconest® (recombinant human C1 esterase inhibitor, or rhC1INH - conestat alfa)
Action
mechanism: Ruconest® (conestat alfa) is a recombinant version of the human protein C1 inhibitor (C1INH). This protein naturally occurs in the human body. It belongs to the class of serine-protease inhibitors or serpins. It regulates several inflammatory pathways in the body by inhibiting certain proteins (proteases) that are part of the human defense system. Deficiency of functional C1 inhibitor leads to excessive activation of the complement system and other immunological and haemostatic pathways, giving cause to angioedema attacks. These attacks are characterized by acute and painful swellings of soft tissues. Administration of C1 inhibitor protein can normalize the immune response and stop the angioedemic attacks.Ruconest® is produced through Pharming’s proprietary technology in milk of transgenic rabbits and is approved in Europe for the treatment of acute angioedema attacks in patients with HAE. Ruconest® has been granted orphan drug designation both for the treatment of acute attacks of HAE and prophylactic treatment of HAE.
Disease: acute attacks of angioedema in patients with Hereditary Angioedema (HAE)
Therapeutic area: Genetic diseases - Rare diseases
Country:
Trial details:
Latest
news: * On November 8, 2013, Pharming Group and Santarus have 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. 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 headacheThe 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 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.
