Paroxysmal Nocturnal Hemoglobinuria Treatment Receives Fast Track Designation

RDR Staff

Apellis Pharmaceuticals announced that their orphan drug APL-2, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) received Fast Track Designation by the FDA. 
APL-2 is designed for patients who experience hemolysis and require RBC transfusions even while taking eculizumab. 
APL-2 currently in two Phase Ib clinical trials. One trial assessing doses APL-2 administered by daily subcutaneous injection (SC) in patients with PNH who have not received the standard of care in the past, and the other is assessing doses of APL-2 as an add on with standard care. 


APL-2 doses of 180mg and 270mg significantly reduced hemolytic activity as early as eight days after the start of dosing, and this inhibition was maintained through the dosing period.

About APL-2

APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer that binds specifically to C3 and C3b, effectively blocking all three pathways of complement activation (classical, lectin, and alternative) with a particularly high potency against the alternative pathway.

About PNH

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder in which red blood cells break apart prematurely. It is an acquired hematopoietic stem cell disorder.
Some hematopoietic stem cells in individuals with PNH are defective and consequently produce defective blood cells. These defective red blood cells of PNH are extremely susceptible to premature destruction by a particular part of a person’s own immune system called the complement system.

About Fast Track Designation 

The FDA’s Fast Track program is designed to facilitate and expedite development and review of new drugs. Through the Fast Track program, a product may be eligible for priority review at the time of BLA and may be eligible to submit sections of the BLA on a rolling basis as data become available.
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