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EMPAVELI® is the first PNH treatment that binds to complement protein C3. It was approved by the Food and Drug Administration in May 2021 for treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH). EMPAVELI is given skin (subcutaneously) by using the Empaveli injector or with an infusion pump 2 times a week.

What is EMPAVELI®?

Empaveli® (previously known as pegacetacoplan) is the first PNH treatment that binds to complement protein C3. It is approved by the Food and Drug Administration for adult patients with paroxysmal nocturnal hemoglobinuria (PNH).   

The effectiveness of Empaveli® was evaluated in a study enrolling 80 patients with PNH and anemia who had been taking eculizumab, a treatment previously approved for PNH. Patients first completed a four-week period during which they received Empaveli® 1,080 mg twice weekly in addition to eculizumab at their previous dose. After the first four weeks, patients were randomly assigned to receive either Empaveli® or their current dose of eculizumab for 16 weeks.  

After 16 weeks, the severity of anemia was compared in the two treatment groups on the basis of hemoglobin concentration (a laboratory measure of anemia). In both treatment groups, the average hemoglobin was 8.7 g/dL at baseline, indicating severe anemia. (Normal hemoglobin values in adult men are 14 g/dL or above; normal values in adult women are 12 g/dL or above.) 
During the 16 weeks of treatment, patients in the Empaveli® group had an average increase in their hemoglobin of 2.4 g/dL. Meanwhile, patients in the eculizumab group had an average decrease in their hemoglobin of 1.5 g/dL.  

It is not known if Empaveli® is safe or effective in children.  

Before taking EMPAVELI®:

Tell your healthcare provider about all your medical conditions, including if you:   

  • have an infection or fever.
  • are pregnant or plan to become pregnant. Empaveli® may harm your unborn baby.
  • Females who are able to become pregnant should have a pregnancy test before starting
  • treatment with Empaveli® .
  • Females who are able to become pregnant should use an effective method of birth  control (contraception) during treatment with EMPAVELI and for 40 days after the final dose.
  • are breastfeeding or plan to breastfeed. It is not known if Empaveli® passes into your breast milk. You should not breastfeed during treatment with Empaveli® and for 40 days after the final dose.

Tell your healthcare provider about all the medicines you take, including prescription and over the-counter medicines, vitamins, and herbal supplements. Empaveli® and other medicines can affect each other, causing side effects.

Know the medicines you take and the vaccines you receive. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.

Who should not receive EMPAVELI®?

Do not take EMPAVELI® if you:

  • are allergic to pegcetacoplan or any of the ingredients in EMPAVELI®. 
  • have not been vaccinated against Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B, unless your healthcare provider decides that urgent treatment with EMPAVELI is needed. 
  • have a serious Streptococcus pneumoniae, Neisseria meningitidis, or Haemophilus influenzae type B infection.

How should patients receive EMPAVELI®?

Your healthcare provider should show you how to prepare and administer Empaveli® before you use it for the first time.

Use Empaveli® exactly as your healthcare provider tells you. Do not use more or less than your healthcare provider tells you to.

Empaveli® is given under the skin (subcutaneously) by using the Empaveli injector or with an infusion pump 2 times a week. If there is an increase in your LDH, an enzyme in your blood, your healthcare provider may tell you to take Empaveli® every 3 days.

If you are changing treatment from ravulizumab to Empaveli®, you should take your starting dose of EMPAVELI no more than 4 weeks after your last dose of ravulizumab.   

If you have PNH and you stop taking Empaveli®, your healthcare provider will need to monitor you closely for at least 8 weeks after stopping Empaveli® . Stopping treatment with Empaveli® may cause a breakdown of red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include:  

  • decreased hemoglobin level in your blood             
  • shortness of breath  
  • tiredness  
  • blood clots o blood in your urine  
  • trouble swallowing
  • pain in the stomach (abdomen)  
  • erectile dysfunction (ED)  

If you miss a dose of Empaveli® , take the missed dose as soon as possible. Take your next dose at your regularly scheduled time.  

What are the possible side effects of EMPAVELI®?   

The most common side effects in people with PNH treated with Empaveli® include injection-site reactions, infections, diarrhea, pain in the stomach (abdomen), respiratory tract infection, viral infection, and tiredness.  

EMPAVELI® can cause serious side effects including:   

  •  Allergic reactions. Allergic reactions can happen during your Empaveli® infusion. Stop your Empaveli® infusion and tell your healthcare provider or get emergency medical care right away if you get any of these symptoms during your Empaveli® infusion:  
    • chest pain  
    • trouble breathing or shortness of breath 
    • swelling of your face, tongue, or throat  
    • feel faint or pass out   

Related Clinical Trials

AAMDSIF does not recommend, endorse, or make any representation about the efficacy, appropriateness or suitability of any drug, treatment or therapy listed on this website. Some therapies listed on our site are considered experimental for the treatment of bone marrow failure diseases. Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding any therapy, and never disregard professional medical advice or delay in seeking it because of something you have read on this website.