2021 FDA Approvals for PNH Treatment: Empaveli for Adults and Ultomiris for Children

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PNH patients may be treated with transfusions, blood cell production hormones, growth factors, transplants, or other treatments for side effects and related conditions.

Three drugs can be used to target the complement system in the treatment of PNH. Each has had success in reducing hemolysis (destruction of red blood cells).

1. Eculizumab (brand name Soliris)
   
   Eculizumab is a monoclonal antibody complement inhibitor targeting complement C5.
   
   Eculizumab is injected into a vein by a healthcare provider.
   
   In the U.S., eculizumab was approved by the FDA for treatment of PNH in March 2007.
   
   
2. Ravulizumab-cwvz (brand name Ultomiris)
   
   Ravulizumab-cwvz is another monoclonal antibody complement inhibitor that targets complement C5.
   
   Ravulizumab is injected into a vein by a healthcare provider.
   
   In the U.S., ravulizumab-cwvz was approved by the FDA for treatment of PNH in adults in December 2018. FDA approval was expanded to infants, children, and adolescents with PNH in June 2021. Due to the higher risk of meningococcal infections, ravulizumab-cwvz is available only through health care providers who are in a registered program called the Risk Evaluation and Mitigation Strategy (REMS).
   
   
3. Pegcetacoplan (brand name Empaveli)
   
   Pegcetacoplan is the newest complement inhibitor for PNH patients. Instead of complement C5, it targets complement C3. Because C3 is "upstream" from C5 (earlier in the complement system), pegcetacoplan can be more successful in preventing hemolysis.
   
   Empaveli is administered by subcutaneous infusion.
   
   In the U.S., pegcetacoplan was approved by the FDA for treatment of PNH in adults in May 2021. Due to the higher risk of meningococcal and other infections, pegcetacoplan is available only through health care providers who are in a registered program called the Risk Evaluation and Mitigation Strategy (REMS).