Pharming Group N.V. has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (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 HAE.
In November 2017, following feedback from FDA on two completed trials of Ruconest for prophylaxis of HAE attacks, Pharming filed a sBLA to expand the approved indication. The Phase 2 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 2 data. Based on their review, the FDA has requested an additional clinical trial to further evaluate the effectiveness of Ruconest in HAE prophylaxis.
“While today’s FDA decision is not what we were anticipating, we look forward to working with the FDA to generate additional clinical data required to enable access for patients to use Ruconest for HAE prophylaxis,” said Dr. Bruno Giannetti, MD PhD, Chief Operations Officer of Pharming.
“We see this as a minor setback. Pharming remains committed to serving the HAE community. We will continue and have the resources to develop new innovative and more convenient administration options of Ruconest for acute treatment and prophylaxis of HAE to improve patient care as outlined in our recent capital market briefing in June,” said Sijmen de Vries, MD, CEO of Pharming.