Rhucin meets Phase III HAE endpoint

Pharming Group N.V. (Euronext:PHARM) and partner Santarus Inc. (NASDAQ:SNTS) reported top-line data showing that a single IV infusion of 50 units/kg Rhucin conestat alfa met the primary endpoint of reducing median time to beginning of symptom relief vs. placebo in the Phase III Study 1310 trial to treat acute attacks of hereditary angioedema (HAE) (90 vs. 152 minutes, p=0.031). The primary endpoint was defined as the time from treatment initiation to the beginning of a persistent beneficial effect based on a patient-reported treatment effect questionnaire of the primary attack location. The positive data trigger a $10 million milestone payment to Pharming from Santarus, which has exclusive commercialization rights to Rhucin in North America. Pharming had an SPA from FDA for the double-blind, U.S. trial, which enrolled 75 patients with HAE.

Pharming plans to submit a BLA to FDA for Rhucin next half. Last year, FDA refused to file a BLA for Rhucin. Pharming subsequently amended 1310's protocol to address issues raised by FDA. The recombinant human complement 1 (C1) esterase inhibitor is approved as Ruconest in the EU to treat acute attacks of HAE (see BioCentury Extra, Feb. 28, 2011). ...