Pharming genetic disorder drug meets trial goals

Hereditary angieoedema (HAE) is a genetic disorder caused by a shortage of C1 inhibitor activity and results in an overreaction of the immune system. The disease is characterized by acute attacks of painful and in some cases fatal swelling of several soft tissues (edema), which may last up to five days when untreated.

The phase III double-blind placebo-controlled study with Rhucin was conducted at several centers across Europe. The trial assessed the safety and efficacy of Rhucin in patients who presented with different sites of acute HAE attacks. In the analysis, data were analyzed from the first 28 patients randomized to either Rhucin or placebo.

Patients receiving Rhucin reported first relief at a median time of 60 minutes compared to 8.5 hours for those patients who received placebo. In addition, patients receiving Rhucin reported minimal symptoms at a median time of 6.1 hours compared with 20.2 hours for patients who received placebo. The analysis demonstrated that 100% of patients in the Rhucin group responded to treatment and that all of these patients experienced sustained relief without a relapse of their attack symptoms.

The positive results from the study have been provided to the Committee for Medicinal Products (CHMP) of the EMEA in support of the marketing authorization for Rhucin in Europe. Data from the analysis were also submitted to the FDA in the US, where an independent placebo-controlled randomized clinical trial assessing the safety and efficacy of Rhucin is expected to be completed in the next few months.