Article | 31 August 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.

Enjoying this article? Have the leading Biopharma news & analysis delivered daily on email by signing up for our FREE email newsletter here. 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. “Following the positive opinion from the European Medicines Agency in June this year, the BLA submission will be the next significant milestone in the development of Rhucin. It demonstrates our commitment to provide global access to this innovative highly effective and safe replacement therapy for HAE patients”, said Rienk Pijpstra, chief medical officer, adding: “The study will further emphasize Rhucin’s benefits for HAE patients such as rapid onset of relief and excellent response rates.” Edison Research comment on US partnering deal Commenting on the news, analysts at Edison Research note that reaching a conclusion on the BLA pathway with the FDA could allow a US partnering deal. An additional clinical study in 50 patients will be run to provide data showing Ruconest’s fast onset of action. While clarity with the FDA is needed for a US deal, a deal may take some time and so may not contribute to 2010 cash. Rather than a deal, Pharming could raise funds for a high profit, direct US sales effort to position Ruconest from 2012 as the preferred HAE acute treatment. It aims to take share from prophylactic treatments. Cash hunting On October 31, 10.9 million euros ($13.8 million) of bonds are due. September cash might be around 9-10 million euros assuming a 5 million-euro European Union approval milestone from Swedish Orphan Biovitrum plus perhaps 1 million euros inventory purchases. R&D will rise as the kidney clinical trials and the new global HAE study get underway. Once the company is solidly funded, it will be in a good strategic position with an imminently approved EU product, new indications about to enter trials and a route to FDA approval of Ruconest, the Edison analysts conclude