Ultomiris (Ravulizumab): A Next-Generation Therapy for Rare Blood and Immune Disorders

Ultomiris (generic name: ravulizumab-cwvz) is a prescription medication used to treat several rare, life-threatening autoimmune and blood disorders. Developed by Alexion Pharmaceuticals, it was approved by the U.S. FDA in 2018 as a longer-lasting alternative to Soliris (eculizumab) — another breakthrough therapy from the same company.

Ultomiris offers patients similar effectiveness with fewer infusions, helping improve both convenience and quality of life.


What Conditions Does Ultomiris Treat?

Ultomiris is approved to treat the following conditions in adults and children:

  • Paroxysmal Nocturnal Hemoglobinuria (PNH): A rare blood disorder where the immune system destroys red blood cells.Atypical Hemolytic Uremic Syndrome (aHUS): A condition that causes small blood clots, kidney damage, and low red blood cell counts.
  • Generalized Myasthenia Gravis (gMG): A chronic autoimmune disorder that weakens voluntary muscles.
  • Neuromyelitis Optica Spectrum Disorder (NMOSD): A rare inflammatory disease affecting the spinal cord and optic nerves.

In each case, Ultomiris helps prevent the body’s immune system from attacking its own cells.


How Ultomiris Works

Ultomiris is a monoclonal antibody that targets a protein in the immune system called C5, part of the complement system.

When overactivated, this system can destroy healthy red blood cells and tissues. By blocking C5, Ultomiris stops the harmful immune attack that drives these diseases — reducing symptoms and preventing serious complications.

Ultomiris works in a similar way to Soliris but stays active in the body much longer, meaning patients need fewer infusions per year.


How It’s Given

Ultomiris is given as an intravenous (IV) infusion by a healthcare professional.

  • The first dose (called a loading dose) is followed by maintenance infusions every 8 weeks — compared to every 2 weeks with Soliris.
  • Each infusion takes about 45 minutes to a few hours, depending on the dose.

Some forms of Ultomiris are also available as subcutaneous injections, which can be given at home under medical guidance.


Common Side Effects

Most side effects are mild and temporary. The most common include:

  • Headache
  • Fatigue
  • Nausea
  • Fever or chills after infusion

Because Ultomiris affects part of the immune system, it increases the risk of meningococcal infections. Patients must receive a meningococcal vaccine before starting treatment.


Benefits of Ultomiris

Clinical studies show that Ultomiris provides:

  • Equivalent efficacy to Soliris in preventing red blood cell destruction and disease relapses
  • Fewer infusions, improving convenience and quality of life
  • Sustained disease control for patients with PNH, aHUS, gMG, or NMOSD

For many patients, Ultomiris offers lasting stability and freedom from the frequent hospital visits required by older treatments.


Final Thoughts

Ultomiris (ravulizumab) is a major step forward in the treatment of rare autoimmune and blood disorders. With its long-acting protection and strong safety profile, it allows patients to focus more on living their lives — not managing constant infusions.

If you or a loved one has PNH, aHUS, gMG, or NMOSD, talk to your healthcare provider to learn whether Ultomiris could be right for you.