How does Soliris work for PNH?

What is the mechanism of action of Soliris?

Soliris works by binding to the complement protein C5. This helps stop the activation of C5a / C5b and prevents formation of terminal complement complex C5b-9. Clinically, Soliris helps to stop red blood cell destruction and symptoms like anemia, fatigue and blood clots in patients living with paroxysmal nocturnal hemoglobinuria (PNH).

In PNH patients are missing a protein that normally protects red blood cells from destruction from terminal complement, a component of the immune system.

What is Soliris approved to treat?

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.

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

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.

Soliris can also used with Voydeya (danicopan), an oral tablet, to treat extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH). Extravascular hemolysis is the breakdown of red blood cells outside of blood vessels in adults with PNH.

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)

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.

How is Voydeya used with Soliris?

In April 2024, Voydeya (danicopan) was approved as add-on therapy to ravulizumab (Ultomiris) or eculizumab (Soliris) for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH).

Voydeya has not been shown to be effective as monotherapy (as a single agent) and should only be prescribed in addition to Soliris or Ultomiris.

The recommended starting dosage of Voydeya oral tablets is 150 mg three times a day, with or without food. Depending on clinical response, your healthcare provider may increase the dose to 200 mg three times a day. Voydeya comes as a 50 mg and 100 mg tablet.

Does Voydeya work differently than Soliris?

Yes, Voydeya is a first-in-class oral Factor D inhibitor that works by selectively inhibiting Factor D, a protein in the complement system that plays a key role in the amplification of the response in extravascular hemolysis (EVH). Soliris is C5 inhibitor and works by binding to the complement protein C5 for the treatment of PNH.

Voydeya is used with Soliris or Ultomiris for PNH to treat the breakdown of red blood cells that takes place outside of blood vessels (extravascular hemolysis) in adults with paroxysmal nocturnal hemoglobinuria (PNH). Voydeya is not used as a single treatment (monotherapy) for extravascular hemolysis in PNH.

About 10% to 20% of adults with PNH experience clinically significant extravascular hemolysis while treated with a C5 inhibitor. Dual complement pathway inhibition at Factor D (with Voydeya) and C5 (with Soliris or Ultomiris) may be an effective treatment approach for the subset of patients with EVH.

It is not known if Voydeya is safe and effective in children.

Learn more: Side effects and warnings with Voydeya

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