FDA refuses to file Rhucin BLA

Shares of Pharming Group N.V. (Euronext:PHARM) and partner Santarus Inc. (NASDAQ:SNTS) fell on Monday after FDA refused to file a BLA for hereditary angioedema (HAE) candidate Rhucin conestat alfa. According to Pharming, the agency said the studies included in the application "did not provide data for a sufficient number of subjects to support the proposed dose of 50 U/kg and lacked prospective validation of the visual analog scale used in measuring the clinical effects of Rhucin." FDA also requested that the results of an ongoing Phase IIIb trial of Rhucin be included in a future BLA submission for the product. The recombinant human complement 1 (C1) esterase is already approved as Ruconest in the EU. ...