FDA lifts partial hold on trials with BCX 9930 in paroxysmal nocturnal hemoglobinuria.- BioCryst Pharma
BioCryst Pharmaceuticals announced that the FDA has lifted its partial clinical hold on the BCX 9930 program. The company will resume enrollment in global clinical trials under revised protocols at a reduced dose of 400 mg twice daily of BCX 9930.
This includes the REDEEM-1 and REDEEM-2 pivotal trials in patients with paroxysmal nocturnal hemoglobinuria (PNH) and the RENEW proof-of-concept trial in patients with C3 glomerulopathy (C3G), immunoglobulin A nephropathy (IgAN) and primary membranous nephropathy (PMN). Clinical evidence and recent laboratory studies have informed the company’s hypothesis that crystals form in the kidneys of some patients. The company believes that lowering the dose to 400 mg and ensuring adequate hydration will dilute the concentration of drug in the urine below the threshold where crystals can form.
REDEEM-1 is a randomized, open-label, active comparator-controlled study of the efficacy and safety of oral BCX9930 monotherapy in PNH patients with an inadequate response to a C5 inhibitor.
REDEEM-2 is a randomized, placebo-controlled study to evaluate the efficacy and safety of oral BCX9930 as monotherapy versus placebo in PNH patients not currently receiving complement inhibitor therapy.
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