FDA Approves First Eculizumab Biosimilar

The FDA approved Bkemv (eculizuamb-aeeb), the first biosimilar to reference Soliris (eculizumab), for the treatment of 2 rare diseases: paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).

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“Many rare conditions are life-threatening, and many do not have treatments,” said Sarah Yim, MD, 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 classified as rare if it affects fewer than 200,000 people in the US. PNH and aHUS are rare diseases that involve the destruction of red blood cells. PNH leads to anemia (low red blood cell count), thrombosis (blood clots), pancytopenia (low counts of red and white blood cells, as well as platelets), and dark urine. Alternatively, aHUS causes anemia, thrombocytopenia (low platelet count), and kidney failure.

Bkemv is a monoclonal antibody that targets the complement C5 protein, inhibiting the activation of the complement system, which is part of the body's immune response. By binding to C5, Bkemv prevents the breakdown of red blood cells in the bloodstream (intravascular hemolysis) in patients with PNH and aHUS.

The interchangeability label will allow pharmacists to exchange Bkemv for Soliris without having to get permission from a physician prior, allowing patients to receive their medications in a more timely manner and avoid delays. Interchangeability labels can also assist in helping pharmacies mitigate supply chain challenges and drug shortages. Bkemv is the 13th biosimilar to be deemed interchangeable.

Similar to Soliris, Bkemv has a boxed warning indicating an increased risk of serious and life-threatening meningococcal infections caused by Neisseria meningitidis, the bacteria responsible for meningitis and other severe infections. Patients should complete meningococcal vaccination before beginning treatment with Bkemv or Soliris. They should also be monitored for early signs and symptoms of meningococcal infections and undergo immediate evaluation if any signs of infection develop.

The biosimilar is scheduled to enter the market on March 1, 2025, after Amgen settled with Alexion, the maker of Soliris, over a patent dispute in 2020.