FDA Approves Bkemv as First Interchangeable Biosimilar to Treat Rare Diseases

The U.S. Food and Drug Administration (FDA) has approved Bkemv (eculizumab-aeeb) as the “first interchangeable biosimilar to Soliris (eculizumab) to treat certain rare diseases.”

Specifically, Bkemv is approved for “the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis” and “the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.”

Rare diseases are defined as those that affect fewer than 200,000 people in the U.S. PNH and aHUS are “rare diseases characterized by the breakdown of red blood cells. PNH results in anemia (low red blood cells), thrombosis (blood clots), pancytopenia (low counts of red blood cells, white blood cells, and platelets) and dark urine, while aHUS results in anemia, thrombocytopenia (low platelets) and kidney failure.”

Bkemv binds to the complement C5 protein to prevent “the breakdown of red blood cells in the bloodstream (intravascular hemolysis) in patients with PNH and aHUS.” It carries the same Box Warning that Soliris does, stating that it increases the risk of “serious and life-threatening meningococcal infections caused by Neisseria meningitidis, the bacteria that causes meningitis and other potentially severe infections.”

Bkemv has no clinically meaningful differences to Soliris. They both have the same safety warnings and are expected to have the same adverse reactions.