FDA issues CRL on Ruconest sBLA

Netherlands-based Pharming Group (Euronext: PHARM) saw its share plunge 29.05% to 0.69 euros by mid-morning today, after it revealed a further setback in its attempt to widen the use of its leading drug.

The US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL regarding the supplemental Biologics License Application (sBLA) for Ruconest (C1 Esterase Inhibitor [recombinant]) to expand the current indication to include prophylaxis in patients with hereditary angioedema (HAE).

In November 2017, following feedback from FDA on two completed trials of Ruconest for prophylaxis of HAE attacks, Pharming filed an sBLA to expand the approved indication. The Phase II studies, an open-label study and a randomized, double-blind, placebo-controlled trial with 4-8 week treatment periods, showed consistent efficacy and safety results.