New recombinant drug is effective in hereditary angioedema
Hereditary angioedema (HAE) is a rare disease caused by a shortage or deficiency of the C1 esterase inhibitor (C1INH) protein. Patients suffer from acute attacks of painful swelling of soft tissues (edema), including limbs, intestines, and larynx, where laryngeal edema can be fatal. A recombinant human C1INH (rhC1INH) was developed to offer an alternative to plasma-derived C1INH.
In the October 2010 issue of The Journal of Allergy and Clinical Immunology (JACI), Zuraw et al report the results of 2 similar but independent, randomized, placebo-controlled studies that were conducted to evaluate the efficacy and safety of 50 and 100 U/Kg of rhC1INH for the treatment of acute attacks of HAE. Both doses significantly reduced the time to beginning of relief of symptoms for all anatomical locations studied. These doses are higher than the recommended dose for plasma-derived C1INH (20 U/Kg), and the response rate for rhC1INH (>90%) was also higher. RhC1INH treatment resulted in an unremarkable safety profile. No dose-related adverse events and no antibody responses against rhC1INH or host-related impurities were observed.
The authors conclude that rhC1INH constitutes a highly effective alternative to plasma-derived C1INH for the treatment of acute angioedema attacks in patients with HAE.
“Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema”
Remarks by Zuraw et al. (JACI October 2010 / Volume 126, No. 4)
The Journal of Allergy and Clinical Immunology (JACI) is the official scientific journal of the AAAAI, and is the most-cited journal in the field of allergy and clinical immunology.