The U.S. Food and Drug Administration (USFDA) approved Bkemv (eculizumab-aeeb) as the first interchangeable biosimilar to Soliris (eculizumab) to treat certain rare diseases. The FDA granted the approval of Bkemv to Amgen Inc.
“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.”
A disease is considered rare if it affects fewer than 200,000 people in the U.S. The conditions Paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) are rare diseases characterized by the breakdown of red blood cells.
According to USFDA, 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, USFDA stated. This binding prevents the breakdown of red blood cells in the bloodstream (intravascular hemolysis) in patients with PNH and aHUS, it added.
“Bkemv, like Soliris, has a Boxed Warning that states that eculizumab products increase the risk of serious and life-threatening meningococcal infections caused by Neisseria meningitidis, the bacteria that causes meningitis and other potentially severe infections. Patients should have completed meningococcal vaccination before starting Bkemv or Soliris, be monitored for early signs and symptoms of meningococcal infections and undergo further evaluation immediately if signs of infection develop,” the US regulatory body said in a statement.
As an interchangeable biosimilar, Bkemv is highly similar with no clinically meaningful differences to Soliris. Bkemv is available only through a restricted program called the Bkemv Risk Evaluation and Mitigation Strategy (REMS). A REMS is a drug safety program that the FDA can require for certain medications with serious safety concerns to help ensure the benefits of the medication outweigh its risks.
Interestingly, Bkemv is the 53rd approved biosimilar in the U.S. The FDA has approved 13 of these as interchangeable biosimilars.