How does Ultomiris compare to Soliris for PNH?

Ultomiris (ravulizumab) and Soliris (eculizumab) are both C5 complement inhibitors approved to treat patients with paroxysmal nocturnal hemoglobinuria (PNH), a blood disease caused by red blood cell destruction. Ultomiris has a longer half-life, meaning it stays in your blood longer and does not have to be dosed as often.

Ultomiris or Soliris can also used with the oral tablet Voydeya (danicopan) to treat extravascular hemolysis (EVH) that may occur in some in up to 20% of patients with PNH. EVH is the breakdown of red blood cells outside of blood vessels. Voydeya is a first-in-class oral Factor D inhibitor with a mechanism that is complementary to the C5 inhibition with Ultomiris or Soliris.

What are Ultomiris and Soliris used to treat?

Soliris is approved to treat:

• patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis (the breakdown of red blood cells)
• patients with atypical hemolytic uremic syndrome (aHUS), a rare chronic blood disease, in adults and children who weigh at least 11 pounds (5 kilograms).
• adult patients with generalized Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody positive
• neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.

Ultomiris is approved to treat:

• adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH).
• adult and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA).
• adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.
• adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive.

Limitations of Use: Neither Ultomiris nor Soliris are indicated for the treatment of patients with Shiga toxin E. coli-related hemolytic uremic syndrome (STEC-HUS).

Both drugs are only available only under a special program to monitor drug safety. You must be registered in the program and understand the risks and benefits of this medicine.

Both Ultomiris and Soliris are manufactured by Alexion Pharmaceuticals.

How does Ultomiris compare to Soliris for paroxysmal nocturnal hemoglobinuria (PNH)?

In studies for PNH evaluated by the FDA, the efficacy of Ultomiris was found to be “non-inferior” to Soliris. A non-inferiority trial attempts to show that the test drug does not perform worse than a comparator drug by more than a small, predetermined margin. The safety profile of Ultomiris was also found to be similar to Soliris.

Ultomiris may be preferred by many healthcare providers and patients because of fewer required intravenous (IV) infusions, less common breakthrough hemolysis, fewer required blood transfusions and a lower overall cost. In addition, studies have reported an improved quality of life in patients receiving Ultomiris for PNH.

What is PNH and what are the symptoms?

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening genetic blood disorder defined by chronic red blood cell destruction, or hemolysis. Patients are missing a protein that protects red blood cells from destruction (hemolysis) from terminal complement.

Soliris and Ultomiris selectively block terminal complement to help prevent hemolysis. Both drugs are classified as C5 complement inhibitors, which reduces the hemolysis that occurs in PNH.

Hemolysis can lead to symptoms such as severe anemia, severe fatigue, pain, shortness of breath, elevated blood pressure in the lung arteries, kidney disease, dark colored urine (hemoglobinuria) and blood clots (thrombosis). Thrombosis can occur throughout the body and result in organ damage and premature death.

Dosing frequency in PNH

Both Ultomiris and Soliris have been shown in clinical studies to be effective in the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

Some clinicians and patients may prefer Ultomiris over Soliris due to fewer intravenous infusions, a major advantage. Intravenous (IV) infusion maintenance doses of Soliris are given every 2 weeks, while IV infusion maintenance doses of Ultomiris are given every 4 or 8 weeks. This could result in up to 20 fewer Ultomiris infusions over one year compared to Soliris.

Ultomiris stays in your blood longer when compared to Soliris, therefore you do not need as many infusions per year.

Ultomiris is also available as a subcutaneous (under the skin) injection for maintenance dosing in adults using an on-body cartridge delivery system. Soliris is only available as an IV infusion.

Ultomiris can be given to adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH), while Soliris is indicated for adults 18 years of age and older for PNH.

Dosing frequency for PNH is as follows:

• Ultomiris: Starting 2 weeks after the initial loading dose, maintenance doses are given every 8 weeks for adults and every 4 or 8 weeks for pediatric patients (depending on body weight).
• Soliris: Administered to adults with PNH as an initial loading dose given weekly for 5 weeks, then as an IV maintenance infusion every 2 weeks.

Learn more:

• Dosing and side effects for Ultomiris (in more detail)
• Dosing and side effects for Soliris (in more detail)

Is there less breakthrough hemolysis with Ultomiris?

A “breakthrough event” during PNH treatment is when at least one new or worsening sign or symptom of hemolysis (red blood cell destruction) occurs along with a rise in lactate dehydrogenase (LDH).

In PNH studies reported by the manufacturer, Ultomiris was shown to have fewer breakthrough events during treatment when compared to Soliris. Breakthrough hemolysis events can lead to symptoms such as fatigue, stomach pain, shortness of breath, darkened urine, and anemia (low red blood cells).

• In one study of people who had never been treated for PNH, 4% of patients (4 out of every 100) receiving Ultomiris had breakthrough hemolysis compared to 10.7% of those who received Soliris.
• In another clinical trial evaluating people who had received prior PNH treatment, no patients (0%) in the Ultomiris group had breakthrough hemolysis compared to 5.1% in the Soliris group.
• After 26-weeks there was no difference observed in patient-reported fatigue between Ultomiris and Soliris groups.

Breakthrough events with Ultomiris may be less when compared to Soliris due to a longer duration of action (half-life) and dosing that is based on weight, among other factors.

Cost of Ultomiris vs. Soliris

Overall treatment costs for Ultomiris are typically lower when compared to Soliris due to fewer infusion and administration costs.

• View the Prices, Coupons and Patient Assistance Programs for Ultomiris
• View the Prices, Coupons and Patient Assistance Programs for Soliris

These prices are for cash paying customers only and are not valid with insurance plans. Prices may vary based upon discounts, location and pharmacy.

Most patients do not pay cash for these medications. Your cost for treatment will depend upon your dose, frequency, length of treatment, and costs to administer your infusions. Your healthcare plan and copay will determine your final costs.

If you need assistance paying for your medicine you can contact Alexion Pharmaceuticals at 1-888-765-4747 or online to determine your eligibility. The Alexion OneSource Copay Program provides financial and insurance information associated with Ultomiris or Soliris, and can help address financial concerns or gaps in coverage.

Can Soliris or Ultomiris be used with Voydeya?

Yes, Soliris or Ultomiris are used with Voydeya in adults to treat the breakdown of red blood cells that takes place outside of blood vessels (extravascular hemolysis) in patients with paroxysmal nocturnal hemoglobinuria (PNH). Extravascular hemolysis occurs in roughly 10% to 20% of patients with PNH.

The recommended starting dose of Voydeya oral tablets is 150 mg three times a day, with or without food. Depending upon your response, your doctor may increase the dose to 200 mg three times a day. Voydeya is available as a 50 mg and 100 mg oral tablet.

Voydeya was approved by the FDA for this use in April 2024. Like Soliris and Ultomiris, Voydeya is manufactured by Alexion Pharmaceuticals.

What is Voydeya?

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 and Ultomiris are C5 inhibitors and work by binding to the complement protein C5 for the treatment of PNH.

Blocking two complement pathways at Factor D (with Voydeya) and C5 (with Soliris) may be an effective treatment approach patients with PNH who experience EVH.

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

This is not all the information you need to know about Soliris, Ultomiris or Voydeya for safe and effective use and does not replace 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.