How does Soliris work for PNH?

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What is the mechanism of action of Soliris?

  • In paroxysmal nocturnal hemoglobinuria (PNH), patients are missing a protein that normally protects red blood cells from destruction from terminal complement, a component of the immune system.
  • Eculizumab, the active ingredient in Soliris, works by strongly binding to the complement protein C5. This binding helps to prevent the activation of C5a and C5b and prevents the generation of the terminal complement complex C5b-9.
  • Soliris can help stop the red blood cell destruction and symptoms like anemia, fatigue and blood clots in patients living with paroxysmal nocturnal hemoglobinuria (PNH).

    • Soliris (eculizumab) is a prescription medicine approved by the FDA to treat paroxysmal nocturnal hemoglobinuria (PNH) in adults 18 years and older. It is a monoclonal antibody and classified as a C5 complement inhibitor. It is not known if it is safe and effective in children.

    For paroxysmal nocturnal hemoglobinuria (PNH) in adults 18 years and older, Soliris is given as an intravenous (IV) infusion into a vein in your arm weekly for 5 weeks, then as a maintenance IV infusion every 2 weeks thereafter.

    What is PNH?

    Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and serious genetic blood disorder that can lead to death. In PNH, patients are missing a protein that protects red blood cells from destruction from terminal complement, part of the immune system.

    Red blood cell destruction can cause symptoms such as:

  • severe anemia
  • severe tiredness
  • pain
  • trouble catching your breath
  • high blood pressure in your lungs
  • kidney disease
  • dark colored urine (hemoglobinuria)
  • blood clots (that may result in organ damage and death)

    • In addition to PNH, Soliris is also approved to treat:

  • Atypical hemolytic uremic syndrome (aHUS), a rare and long-term blood disease in adults and children. Soliris is not for use in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STECHUS)
  • Myasthenia Gravis in certain adults
  • Neuromyelitis optica spectrum disorder (NMOSD) in certain adults

    • How long does it take for Soliris to work in PNH?

    At the end of a 26-week study, patients with paroxysmal nocturnal hemoglobinuria (PNH) treated with IV Soliris had significantly reduced (p< 0.001) red blood cell destruction (hemolysis). Some reductions in hemolysis occurred within one week of starting treatment.

    This led to improvements in anemia as shown by increased hemoglobin levels and reduced need for blood transfusions compared to patients who received a placebo (an inactive agent). In addition, after 3 weeks of treatment, patients reported less fatigue and improved health-related quality of life.

    The effect of Soliris on blood clotting events in the 26-week study could not be determined due to limitations on study size and duration.

    In a long-term extension study in 187 patients receiving Soliris, all patients maintained a reduction in hemolysis over a 10 to 54 month period. There were fewer clotting events than in the same time period prior to treatment, but most patients were also receiving anticoagulant (“blood-thinning”) medicines. Researchers did not study the effect of stopping the anticoagulant in patients also receiving Soliris.

    This is not all the information you need to know about Soliris (eculizumab) for safe and effective use and does not take the place of your doctor’s directions. Review the full Soliris information and discuss this information and any questions you have with your doctor or other health care provider.

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