FDA approves Amgen's Soliris biosimilar

The Food and Drug Administration approved Amgen’s Bkemv (eculizumab-aeeb) as the first interchangeable biosimilar to AstraZeneca's Soliris (eculizumab) to treat certain rare diseases. Bkemv is approved for the following treatment indications, which are also currently approved for Soliris:

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.

"Many rare conditions are life-threatening, and many do not have treatments," said Sarah Yim, director of the Office of Therapeutic Biologics and Biosimilars in the FDA's Center for Drug Evaluation and Research. "The FDA is committed to help facilitate the development of safe and effective interchangeable biosimilar treatments that can expand access for individuals with rare diseases whose current treatment options are limited."

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A disease is considered rare if it affects fewer than 200,000 people in the U.S. The conditions 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 is a monoclonal antibody that binds to the complement C5 protein and inhibits activation of the complement system, a part of the body's immune system. This binding prevents the breakdown of red blood cells in the bloodstream (intravascular hemolysis) in patients with PNH and aHUS.

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