FDA Fast Tracks Rare Hematological Disorder Treatment
Apellis has announced that the Food and Drug Administration (FDA) has granted Fast Track designation to APL-2, a novel drug candidate studied for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Specifically, APL-2 is intended for PNH patients who continue to experience hemolysis and require red blood cell (RBC) transfusions despite receiving therapy with eculizumab.
PNH is a very rare, life-threatening and debilitating hematological disorder. APL-2 is a complement C3 inhibitor and a synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer. It effectively blocks all three pathways of complement activation (classical, lectin, and alternative) with a particularly high potency against the alternative pathway. This mechanism may have the potential to control symptoms and modify the underlying disease in patients with PNH.
Two Phase 1b clinical trials are currently ongoing to evaluate APL-2: PADDOCK and PHAROAH. They are assessing the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary efficacy of single and multiple doses of APL-2 administered by subcutaneous (SC) injection as either an add-on to standard of care or in patients who have not previously received standard of care.
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