Pharming Group NV concludes End-of-Phase 2 interactions with the US FDA for the expanded use of RUCONEST in HAE patients.
M2 PHARMA-September 11, 2017-Pharming Group NV concludes End-of-Phase 2 interactions with the US FDA for the expanded use of RUCONEST in HAE patients
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Pharmaceutical company Pharming Group NV (Euronext Amsterdam:PHARM) said on Friday that it has completed its End-of-Phase 2 interactions with the US Food and Drug Administration (FDA) with respect to RUCONEST for the treatment of Hereditary Angioedema (HAE) patients, a rare genetic disorder characterized by spontaneous and recurrent episodes of swelling (edema attacks).
During the interactions, the company provided the US FDA with the results of two completed Phase 2 trials of RUCONEST for the prophylaxis of HAE attacks as well asa randomized, double-blind, placebo-controlled trial and an open-label study. The two studies enrolled 56 patients and showed consistent efficacy and safety results.
In Q4 of this year, the company will file with the US FDA a BLA efficacy supplement (sBLA) to include routine prophylaxis against angioedema attacks in adolescent and adult patients with Hereditary Angioedema (HAE) as an expanded indication for RUCONEST based on the feedback from the US FDA.
The company said RUCONEST is a recombinant C1 esterase inhibitor (C1-INH) indicated for the treatment of acute attacks in adult and adolescent patients with HAE. It granted US FDA's approval for HAE in July 2014. RUCONEST addresses the cause of HAE attacks by increasing C1-INH in the plasma to normal levels and by stopping the production of kallikrein, an enzyme that activates bradykinin and causes blood vessels to leak.
HAE is caused by a deficiency of the C1 esterase inhibitor protein and because defective C1-Inhibitor does not perform its regulatory function, a biochemical imbalance can occur and produce unwanted peptides that induce the capillaries to release fluids into surrounding tissue, causing swelling or edema concluded the company.
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|Date:||Sep 11, 2017|
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