Pharming plans to file Rhucin BLA with US FDA by end 2010

Netherlands-based biotechnology company Pharming Group NV (NYSE Euronext: PHARM) says that it intends to submit the Biologic License Application the US Food and Drug Administration to obtain marketing approval for Rhucin (recombinant human C1 esterase inhibitor) for the treatment of acute angioedema attacks in patients with hereditary angioedema (HAE). Following pre-BLA discussions with the FDA, Pharming says it is preparing the BLA dossier for submission towards the end of this year but no later than January 2011.

The BLA will be based on the data of the European Marketing Authorization Application (MAA), on which the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion in June of this year. The company says the BLA will be updated with patient data collected since the completion of the MAA package and will include additional analyses requested by the FDA. The BLA dossier will include data on over 500 administrations in over 150 patients, demonstrating the safety and efficacy of Rhucin for the treatment of HAE attacks.

To further strengthen Rhucin's competitive profile, Pharming is preparing to initiate a Phase IIIB/IV study. In this global multicenter randomized placebo-controlled study, focusing on 'time to onset of relief' of HAE symptoms, 50 patients will either receive 50U/kg Rhucin (also known as Ruconest) or placebo.