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

Ruconest is currently approved to treat acute attacks in patients with HAE. The Company filed the sBLA last November following FDA feedback on two completed Phase 2 trials of Ruconest for prophylaxis of HAE attacks. One trial was open-label, while the other was randomized, double-blind, and placebo-controlled.  

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According to the CRL, the Agency has requested an additional clinical trial to further evaluate the effectiveness of the treatment for prophylaxis in HAE patients. 

“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, COO of Pharming.

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