Biotech company Pharming Group NV ("Pharming" or "the Company") (NYSE Euronext: PHARM) today announced that it has submitted the Marketing Authorization Application (MAA) for Rhucin for the treatment of acute attacks of Hereditary Angioedema (HAE) to the European Medicines Agency (EMEA).
In this new MAA, Pharming has followed up on and addressed concerns raised by the CHMP during the former evaluation procedure in 2007. In particular, the size of the clinical database has been significantly expanded, additional clinical efficacy and safety data have been collected and analyzed, and an analysis of immunogenicity data after (repeat) treatment with Rhucin has been completed.
Efficacy of Rhucin was demonstrated in two randomized controlled studies and is supported by four open label studies, including the successful treatment of potentially life-threatening laryngeal attacks. In the placebo controlled clinical studies a total of 70 patients were randomized to Rhucin (100 U/kg or 50 U/kg body weight) or placebo control treatment. Patients with active Rhucin treatment responded rapidly to treatment, with statistically significant and clinically relevant shorter time to onset of relief and time to minimal symptoms compared to placebo. No relapses of attacks were recorded. Superiority over placebo was also consistently supported by exploratory endpoints, sensitivity analyses and subgroup analyses. Additional data on 155 attacks in 79 patients collected in the ongoing open-label studies furthermore confirmed Rhucin's efficacy.
The updated safety dataset comprising 405 administrations in 164 subjects, includes 14 patients with at least five repeat administrations, Rhucin continued to be well tolerated, the adverse event profile observed in the controlled studies, was similar to that of placebo, and there were no significant infusion site reactions such as pain. The immunogenicity analysis of 621 plasma samples covering 217 treatments in 119 patients showed a reassuring immuno-safety profile, without evidence for induction of neutralizing antibodies against Rhucin, nor for induction of allergies.