New data support efficacy of Ruconest across all HAE attack locations
Leiden, The Netherlands, 23 May 2011. Pharming Group NV ("Pharming" or "the Company") (NYSE Euronext: PHARM) announced today that new clinical data from open-label studies on their recombinant human C1 inhibitor (rhC1INH; conestat alfa; Ruconest(TM) in Europe, Rhucin® in other countries) 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.
Dr. Pijpstra, Chief Medical Officer at Pharming commented: "These new data support that Ruconest is an effective novel therapy for the treatment of acute HAE attacks, including those potentially life-threatening attacks involving the upper airways. In addition, the European open label efficacy data suggest that although a 50 U/kg dose is recommended to achieve optimal efficacy, many attacks respond well to a single vial dose of 2100 U."
About Pharming Group NV
Pharming Group NV is developing innovative products for the treatment of unmet medical needs. Ruconest(TM) (Rhucin® in non-European territories) is a recombinant human C1 inhibitor approved for the treatment of angioedema attacks in patients with HAE in all 27 EU countries plus Norway, Iceland and Liechtenstein, and is distributed in the EU by Swedish Orphan Biovitrum (STO: SOBI). The product is also under development for follow-on indications, i.e. antibody-mediated rejection (AMR) and delayed graft function (DGF) following kidney transplantation. The advanced technologies of the Company include innovative platforms for the production of protein therapeutics, technology and processes for the purification and formulation of these products. Additional information is available on the Pharming website, www.pharming.com<http://www.pharming.com/>.
This press release contains forward looking statements that involve known and unknown risks, uncertainties and other factors, which may cause the actual results, performance or achievements of the Company to be materially different from the results, performance or achievements expressed or implied by these forward looking statements.
Contact: Rienk Pijpstra, CMO, Sijmen de Vries, CEO, T: +31 (0)71 52 47 400
Press release (PDF)<http://hugin.info/132866/R/1517766/454313.pdf>
Posted: May 2011