Salix Pharmaceuticals and Pharming have announced US Food and Drug Administration (FDA) approval of Ruconest for treatment of acute angioedema attacks in adult and adolescent patients with hereditary angioedema (HAE).
The FDA has approved the company’s biologics licence application for Ruconest, a C1 esterase inhibitor [recombinant], based on a Phase III trial (RCT) that included an open-label extension (OLE) phase and is supported by results of two additional RCTs and two additional OLE studies.
The pivotal RCT and OLE studies included 44 patients who experienced 170 HAE attacks. The primary efficacy endpoint was the time to beginning of symptom relief, assessed using patient-reported responses to two questions about the change in overall severity of their HAE attack symptoms after the start of treatment.
Headache, nausea, and diarrhoea were the most common adverse reactions, while anaphylaxis was the serious adverse reaction in clinical studies of Ruconest.
Pharming CEO Sijmen de Vries said: "The approval of Ruconest in the US is a very significant milestone for Pharming.
"For many years we have strived to make Ruconest, the first recombinant replacement therapy for C1Inhibitor deficiency, available to the HAE patient community in the US, because we were aware of the great value and benefit this product adds to patients’ lives.
"Today we are proud to have achieved this goal in the US."
Ruconest is manufactured by Pharming in the Netherlands and Salix Pharmaceuticals has licensed exclusive rights from Pharming to commercialise the drug in North America.
Salix Pharmaceuticals currently plans on making Ruconest accessible to patients later this year.