Complete Response Letter for Ruconest sBLA for expanded indication in hereditary angioedema.- Pharming.

Pharming has received a Complete Response Letter (CRL) from the FDA 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. In January 2018, FDA deemed the application as sufficiently complete to permit a substantive review of the Phase II data. Based on their review, the FDA has requested an additional clinical trial to further evaluate the effectiveness of Ruconest in HAE prophylaxis.

Comment: Rhucin (the European name of the drug) was approved in the EU in October 2010 and is marketed there by Swedish Orphan Biovitrium.