August 26, 2010: Biotech company Pharming Group NV intends to submit a Biologic License Application (BLA) to the FDA to obtain marketing approval for Rhucin for the treatment of acute angioedema attacks in patients with hereditary angioedema (HAE). Following pre-BLA discussions with the FDA, Pharming 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 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 more than 500 administrations in more than 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 3B/4 study. In this global multi-centre randomised placebo-controlled study, focusing on ‘time to onset of relief’ of HAE symptoms, 50 patients will either receive 50U/kg Rhucin 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 Dr Rienk Pijpstra, chief medical officer at Pharming. “The study will further emphasise Rhucin’s benefits for HAE patients, such as rapid onset of relief and excellent response rates.”
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