USFDA grants approval of Bkemv to Amgen

The US Food and Drug Administration approved Bkemv (eculizumab-aeeb) as the first interchangeable biosimilar to 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, Office of Therapeutic Biologics and Biosimilars, 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.” 

The conditions PNH and aHUS are rare diseases characterised by the breakdown of red blood cells. PNH results in anaemia (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 anaemia, 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. 

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.

As an interchangeable biosimilar, Bkemv is highly similar with no clinically meaningful differences to Soliris. Bkemv has the same safety warnings and is expected to have the same adverse reactions as Soliris. The most frequently reported adverse reactions in the PNH randomised trial for Soliris (≥10 per cent overall and greater than placebo) are headache, nasopharyngitis (common cold), back pain and nausea. The most frequently reported adverse reactions in aHUS single arm prospective trials for Soliris (≥20 per cent) are headache, diarrhoea, hypertension, upper respiratory infection, abdominal pain, vomiting, nasopharyngitis, anaemia, cough, swelling of lower legs or hands, nausea, urinary tract infections and fever. 

Bkemv is available only through a restricted program called the Bkemv Risk Evaluation and Mitigation Strategy (REMS).