Supplemental Biologics License Application to FDA

Pharming Group N.V. has submitted a supplemental Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for Ruconest [Recombinant Human C1 Esterase Inhibitor/ conestat alfa] for routine prophylaxis to prevent attacks in adult and adolescent patients with HAE.

The submission includes data from two completed trials of Ruconest for the prophylaxis of HAE attacks: a randomized, double-blind, placebo-controlled trial and an open-label study. The two studies enrolled a total of 56 patients and showed consistent efficacy and safety results.

Dr. Bruno Giannetti, MD PhD, COO of Pharming, commented: “HAE patients in the US are currently facing a shortage of plasma-derived C1 inhibitor used to prevent attacks. We understand that this supply disruption has had serious consequences for them, including additional stress, disease-related complications, and hospitalizations. We look forward to working with FDA and potentially providing these patients an alternative and plasma free option for HAE prophylaxis.”
(Source: Pharming)

2017-11-28T00:15:52+00:00