BioCentury
ARTICLE | Clinical News

Rhucin: Phase III data

November 12, 2012 8:00 AM UTC

Top-line data from the double-blind, U.S. Phase III Study 1310 trial in 75 patients with HAE showed that a single IV infusion of 50 units/kg Ruconest met the primary endpoint of reducing median time to beginning of symptom relief vs. placebo (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. Patients receiving Ruconest reported adverse events of sneezing, procedural headache, back pain, skin burning sensation, an increase in fibrin D-dimer and lipoma. No patient experienced a thromboembolic event, anaphylaxis or neutralizing antibodies to C1 esterase inhibitor. There was 1 serious adverse event reported of abdominal hernia, which was deemed not related to Ruconest.

The trial, for which Pharming has an SPA from FDA, included a 90-day follow-up period during which patients could receive open-label treatment for a subsequent attack. Pharming plans to submit a BLA to FDA for Ruconest next half. The positive data trigger a $10 million milestone payment to Pharming from Santarus Inc. (NASDAQ:SNTS, San Diego, Calif.), which has exclusive commercialization rights to Ruconest in North America. ...