Biotech company Pharming Group NV announced positive results from ongoing open-label studies with recombinant human C1 inhibitor (Rhucin) for the treatment of acute attacks of Hereditary Angioedema (HAE).
The positive results are from the treatment of 123 acute HAE attacks in 64 patients with different doses of Rhucin in the ongoing European and North American open-label studies. Rhucin has been consistently safe and effective in patients receiving up to nine treatments with no evidence of decreased response to the study drug. Importantly, all seven serious laryngeal attacks treated in these studies responded rapidly to Rhucin. The open-label data are consistent with findings from Pharming's two randomized, double-blind, placebo-controlled studies of Rhucin with a median time to onset of relief of one hour and a median time to minimal symptoms of four hours.
Dr. Bruno Giannetti, chief operations officer at Pharming, commented: "We are very pleased with these positive results with Rhucin. They confirm the clinical benefits of Rhucin, also in repeat treatment, without showing any adverse reactions or immune responses. Furthermore, the total number of treatments has substantially increased, including the successful treatment of serious potentially life-threatening laryngeal attacks. We very much appreciate the support of the investigators and patients with HAE with these clinical trials. These studies are an important contribution to making Rhucin available for HAE patients worldwide."
The results from open-label studies confirm Rhucin to be safe and effective in the repeat treatment of acute HAE attacks at different dosage regimens. No clinically relevant adverse reactions were reported from these studies. The open-label treatments represent a substantial increase to the Rhucin clinical database and will be used to support planned regulatory submissions.
Rhucin (recombinant human C1 esterase inhibitor) is a human protein developed through Pharming's proprietary technology where the human protein is expressed in milk of transgenic rabbits. Rhucin is currently under development for treatment of patients with acute attacks of Hereditary Angioedema (HAE). HAE is a human genetic disorder caused by a shortage of C1 inhibitor activity and results in an overreaction of the immune system. The disease is characterized by acute attacks of painful and in some cases fatal swelling of several soft tissues (edema), which may last up to five days when untreated.
Pharming Group NV is developing innovative products for the treatment of genetic disorders, ageing diseases, specialty products for surgical indications, intermediates for various applications and nutritional products.