How does Ultomiris compare to Soliris for PNH?

Official answer

by Drugs.com

Key Points

Ultomiris (ravulizumab) and Soliris (eculizumab) are both prescription medicines approved by the FDA to treat patients with paroxysmal nocturnal hemoglobinuria (PNH).

Both product are classified as C5 complement inhibitors, but Ultomiris has a longer half-life, meaning it stays in your blood longer and does not have to be dosed as often. View dosing here.

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.

However, 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 are Ultomiris and Soliris used to treat?

Soliris is approved to treat:

patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis

patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy

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.

Limitations of Use: Ultomiris is not 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.

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 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.

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 detail)

Dosing and side effects for Soliris (in 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 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.

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 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

Ultomiris costs about $6,695 per vial, compared with $6,820 per vial for Soliris. However, overall treatment costs for Ultomiris are lower when compared to Soliris due to fewer infusion and administration costs.

The cost for Ultomiris intravenous solution (100 mg/mL) is around $6,695 for a supply of 3 milliliters.

The cost for Soliris intravenous solution (10 mg/mL) is approximately $6,820 for a supply of 30 milliliters.

These prices are for cash paying customers only and are not valid with insurance plans. These prices can 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 health plan and copay will determine your final costs.

If you need assistance paying for your medicine you can contact Alexion at 1-888-765-4747 or online to determine your eligibility. The Alexion OneSource Copay Program provides financial assistance by covering your out-of-pocket medication and infusion costs associated with Ultomiris or Soliris, if you are eligible.

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

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