Date: 2011-05-23
Type of information:
phase: 3
Announcement: results
Company: Pharming (The Netherlands)
Product: Ruconest® (Rhucin® in non-European territories - conestat alfa)
Action
mechanism: Ruconest® is a recombinant version of the human protein C1 inhibitor (C1INH). It is produced through Pharming’s proprietary technology in milk of transgenic rabbits.
Disease: acute Hereditary Angioedema (HAE) attacks
Therapeutic area: Rare diseases - Genetic diseases
Country:
Trial details:
Latest
news: Pharming Group NV has announced that new clinical data from open-label studies on their recombinant human C1 inhibitor was presented during the 7th C1INH deficiency workshop in Budapest, Hungary, 20-22 May 2011.
New data was presented on 194 treatments with Ruconest® / Rhucin® using a fixed dose of one vial (2100 U) or more at the investigator's discretion, in 57 patients with acute Hereditary Angioedema (HAE) attacks. This open-label study was an extension of one of the two pivotal randomized controlled studies which formed the basis for approval in Europe. The majority of acute Angioedema attacks (63%) were treated with a single dose of Ruconest® (2100 U). Use of more than one dose was more common during attacks at oro-facial-pharyngeal-laryngeal locations (57%) than during abdominal (26%) and peripheral (37%) attacks. The median time to the beginning of relief of symptoms across treatments, was approximately 60 minutes with an overall response rate of 87% and no relapses. No product related adverse reactions were observed during this study.
Results were also presented for 53 potentially life threatening acute angioedema attacks involving the upper airways. The median time to the beginning of relief of symptoms for these upper airway attacks was 76 minutes (95% confidence interval, 62; 120 min) and the median time to the beginning of relief of symptoms was 265 minutes (95% confidence interval, 240; 720 min). These results are consistent with previously reported results for other anatomical locations. The overall response rate in treating these severe attacks was 100%. There were no treatment failures, nor relapses reported, and Ruconest was generally safe and well tolerated. The safety dataset of Ruconest® / Rhucin® now includes a total of 714 administrations in 190 subjects.
