US FDA accepts Ruconest filing from Santarus and Pharming

US drugmaker Santarus (Nasdaq: SNTS) and Netherlands-based Pharming Group (NYSE Euronext: PHARM) say that the US Food and Drug Administration has accepted for filing the Biologics License Application for the investigational drug Ruconest (recombinant human C1 esterase inhibitor) 50IU/kg. Ruconest is approved in Europe for the treatment of acute angioedema attacks in patients with HAE.

Santarus and Pharming are seeking U.S. marketing approval of Ruconest for the treatment of acute angioedema attacks in patients with hereditary angioedema (HAE). The FDA indicated that, as part of its review, it plans to present the BLA to the Blood Products Advisory Committee. Under the Prescription Drug User Fee Act (PDUFA) guidelines, the companies expect the FDA will complete its review or otherwise respond to the Ruconest

The safety and efficacy of Ruconest for the treatment of HAE attacks were evaluated in a clinical program that included a Phase III randomized placebo-controlled study conducted under a Special Protocol Assessment agreement with the FDA. The Ruconest clinical program also included two additional randomized placebo-controlled studies and several open label treatment studies.