FDA Fast Tracks Rare Hematological Disorder Treatment

Paroxysmal nocturnal hemoglobinuria is a very rare, life-threatening hematological disorder
Paroxysmal nocturnal hemoglobinuria is a very rare, life-threatening hematological disorder

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. 

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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.

For more information visit Apellis.com.