Ravulizumab (ravulizumab) — Uses, Dosage, Side Effects & Safety

Drug class: Complement inhibitor (monoclonal antibody)

Overview

Ravulizumab is a prescription medication used to treat rare, life-threatening blood disorders such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in adults and children. It works by blocking a part of your immune system called complement C5, which helps prevent the destruction of red blood cells and reduces the risk of blood clots and organ damage. This medicine is given as an intravenous (IV) infusion, usually every 8 weeks after an initial loading dose. Ravulizumab can help improve symptoms like fatigue, anemia, and kidney problems associated with these conditions. Patients should be aware that it can increase the risk of certain infections, especially meningococcal infections, so vaccination and close monitoring are important.

Therapeutic uses

  • Paroxysmal nocturnal hemoglobinuria (PNH)
  • Atypical hemolytic uremic syndrome (aHUS)
  • Generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive
  • Neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive

Common side effects

  • Headache
  • Nausea
  • Diarrhea
  • Upper respiratory tract infection
  • Meningococcal infection
  • Infusion-related reactions
  • Anaphylaxis

Frequently Asked Questions about Ravulizumab

What conditions does ravulizumab treat?

Ravulizumab is used to treat paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in adults and children.

How is ravulizumab given?

It is given as an intravenous infusion by a healthcare professional, typically every 8 weeks after an initial loading dose.

What are the most serious risks of ravulizumab?

The most serious risk is life-threatening meningococcal infection. Vaccination and close monitoring are essential.

Can I receive vaccines while on ravulizumab?

Avoid live vaccines during treatment. Ensure you are vaccinated against meningococcal infection before starting therapy.

What should I do if I miss a dose?

Contact your healthcare provider as soon as possible to reschedule your infusion. Do not attempt to self-administer.

Is ravulizumab safe during pregnancy or breastfeeding?

There is limited data on safety during pregnancy or breastfeeding. Use only if the potential benefit justifies the potential risk.

Are there alternatives to ravulizumab?

Yes, other complement inhibitors such as eculizumab may be considered. Your doctor will determine the best option for you.

Educational information only. Always consult a qualified healthcare professional before starting, stopping, or changing any medication.