Pharming, Santarus File BLA

Pharming Group NV and Santarus, Inc. have submitted a BLA to the FDA to obtain marketing approval for Rhucin (recombinant human C1 inhibitor) for the treatment of acute angioedema attacks in patients with Hereditary Angioedema (HAE).

The safety and efficacy of Rhucin for the treatment of HAE attacks were evaluated in two randomized placebo-controlled studies and are supported by four open label treatment studies. Both placebo-controlled clinical studies showed statistically significant and clinically relevant improvement in the primary endpoint of time to beginning of relief of symptoms at Rhucin dosage strengths of 50 U/kg and 100 U/kg compared to placebo.

In October 2010, Pharming received Marketing Authorization for Ruconest (Rhucin in non-European countries) for the treatment of acute HAE in the EU. Pharming has updated the clinical dataset reviewed and approved by the European Medicines Agency with additional patient data and analyses. In total, the BLA dossier includes nine clinical studies covering 714 administrations in 190 subjects.

Santarus has licensed certain exclusive rights from Pharming to commercialize Rhucin in North America for the treatment of acute attacks of HAE and other future indications. Under the terms of the license agreement, a $5 million milestone is payable to Pharming upon FDA acceptance for review of the BLA for Rhucin.