BRIEF—sBLA for Pharming's Ruconest

Netherlands-based Pharming Group (Euronext: PHARM) has submitted a supplemental Biologics License Application (sBLA) to the US 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 hereditary angioedema (HAE).

Ruconest has already been approved by the FDA for the treatment of acute attacks in adult and adolescent patients with HAE. If the sBLA receives the nod, it will become the only product on the market to offer both acute and prophylactic treatment options.

Bruno Giannetti, chief operations officer of Pharming, said: “HAE patients in the USA 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.”