If you’re living with paroxysmal nocturnal hemoglobinuria (PNH), does your current treatment plan meet your needs? Are the side effects acceptable? If you’ve ever wished for more PNH treatment options, it’s a good time to read up on promising options currently being studied.

Researchers are working to develop new PNH therapies and treatment plans. These updated strategies may work better than previous treatments, lead to milder side effects, or help improve quality of life. New treatment options may also help people with PNH live longer.

Whether you were recently diagnosed or if you’ve been treating PNH for many years, it’s best to understand all of your therapy options. Talk to your health care team if you’re interested in learning more about new treatments and exploring whether a new option may work better for you.

New PNH Drugs

Current PNH treatments like eculizumab (Soliris) block your complement system — the part of your immune system that attacks your red blood cells (RBCs) when you have PNH. Eculizumab, which was approved by the U.S. Food and Drug Administration (FDA) in 2007, blocks a protein in your complement system called factor C5. In recent years, the FDA has also approved biosimilars for eculizumab. This means they’re considered interchangeable with Soliris. Approved biosimilars for eculizumab include eculizumab-aeeb (Bkemv) and eculizumab-aagh (Epysqli).

However, newer medications may work in different ways to block processes involved in PNH and treat its symptoms.

Iptacopan (Fabhalta) for Adults With PNH

Iptacopan (Fabhalta) is a new oral PNH medication that was approved by the FDA in December 2023. It blocks a complement system protein called complement factor B. It’s the first oral medication for treating PNH in adults.

In a study of nearly 100 people with PNH, researchers found that iptacopan helped reverse hemolysis. Additionally, most participants who took the medication no longer needed blood transfusions.

Iptacopan may also provide other benefits. People taking this drug may be more likely to have normal levels of RBCs, which may mean fewer symptoms and a better quality of life.

Pegcetacoplan (Empaveli) for Adults With PNH

In May 2021, the FDA approved a PNH medication called pegcetacoplan, sold under the brand name Empaveli. It’s a monoclonal antibody, which means it’s a type of protein made in a laboratory. Pegcetacoplan is the first PNH treatment to block complement factor C3 — a different complement protein than the ones targeted by eculizumab and iptacopan.

Studies have found that pegcetacoplan works well to reverse PNH. In one clinical trial, researchers looked at the effectiveness of pegcetacoplan by measuring hemoglobin levels. Hemoglobin is released into the blood and removed in the urine when RBCs break apart, so people with untreated PNH often have high levels of this protein in their urine, hence the name “hemoglobinuria.”

However, in a study published in The New England Journal of Medicine, researchers found that when people took pegcetacoplan, their hemoglobin levels increased more than in those who used eculizumab. This was because pegcetacoplan caused less destruction of red blood cells.

Additionally, none of the patients in this study developed meningitis, an infection of the membranes covering the spinal cord and brain. People treated with eculizumab have previously experienced this infection, and they need to get vaccinated to prevent it.

In some cases, people with PNH also need blood transfusions (a procedure in which your doctor gives you donated blood). However, the above study reported that 85 percent of people who took pegcetacoplan no longer needed transfusions, compared to 15 percent of those who used eculizumab.

This study found that some people with PNH have fewer symptoms when they switch to pegcetacoplan after using eculizumab. Other studies have also reported positive outcomes when those who have never used PNH medications start taking pegcetacoplan.

Pegcetacoplan is generally safe. However, it can cause side effects like diarrhea and leads to infections at a similar rate as eculizumab.

Overall, these studies show that pegcetacoplan for PNH may be a helpful option for many people.

Ravulizumab (Ultomiris) for Adults and Children With PNH

Ravulizumab, sold under the brand name Ultomiris, is a medication that works similarly to eculizumab — it blocks complement factor C5. Studies have found that both paroxysmal hemoglobinuria drugs are similarly safe and effective. Eculizumab is given every two weeks, and ravulizumab is taken every two months.

In 2018, the FDA approved ravulizumab as a treatment for adults, so it’s been available for a few years for those over the age of 18. In 2021, the FDA also approved the medication for children and teens with PNH. It can now be used by anyone over the age of 1 month. Research has shown that ravulizumab may work better than eculizumab to improve the well-being of children with this condition.

Crovalimab-akkz (PiaSky) for Ages 13 and Up

In 2024, the FDA approved crovalimab-akkz (PiaSky) to treat PNH in people aged 13 and older who weigh at least 88 pounds (40 kilograms). Crovalimab-akkz is a C5 inhibitor, and it works in a similar way as eculizumab. One important difference between the medications is the way they’re administered. While eculizumab is infused through an IV infusion (into a vein), crovalimab-akkz is given as a subcutaneous (under the skin) injection every four weeks, after loading doses.

In a clinical trial, study participants took either crovalimab-akkz or eculizumab, and results were compared. Crovalimab-akkz worked as well as eculizumab to control hemolysis and prevent the need for blood transfusions.

Bone Marrow Transplantation

PNH affects the cells in your bone marrow that make new blood cells. Mutations (changes) in these cells lead to damaged RBCs that are easily killed by the complement system. Allogeneic bone marrow transplants are procedures in which abnormal cells are killed and replaced with healthy cells. This treatment is the only possible cure for PNH. Allogeneic bone marrow transplants are sometimes used to treat PNH and related conditions like aplastic anemia, but they aren’t a good fit for everyone because they can lead to life-threatening side effects.

Bone marrow transplants have been used for many years as a PNH treatment. However, researchers continue to study this procedure to determine when it’s most helpful. In recent years, they have concluded that certain factors lead to better results after transplantation. You’re more likely to live longer after this procedure if, before getting a transplant, you:

• Are under the age of 30
• Have needed 20 or fewer blood transfusions
• Didn’t have hemolysis (your RBCs weren’t getting destroyed)

When deciding whether a bone marrow transplant is a good option for you, your doctor may consider the above factors.

Drugs Being Studied for the Treatment of PNH

Additional therapies are also being tested in clinical trials. If studies show that these medications are safe and effective, they may be approved by the FDA and become widely available to those with PNH. Some new medications target the same complement proteins as existing drugs, while others block different aspects of the complement system.

Complement Factor D

Some potential PNH treatments target a protein called complement factor D. A couple of early studies reported that the medications danicopan and BCX9930 may help raise hemoglobin levels for those with PNH, although more research is needed.

Clinical Trials

You may be able to receive these therapies or others under investigation if you join a clinical trial. These studies aim to analyze the positive and negative effects of new medications, drug doses, or treatment plans. If you’re interested in participating in a clinical trial, ask your doctor if they can help you find PNH trials you might be eligible to join in your area.

Talk to Your Doctor About New PNH Treatments

If you’re not happy with your current PNH treatment plan, the first step is to talk to your health care team. Alternatively, your doctor may suggest switching treatments if they think a different therapy may be better for your needs.

Changing to a new treatment is worth discussing with your PNH specialist if:

• Your current medication isn’t helping as much as expected (for example, if you need frequent blood transfusions or if your hemoglobin levels continue to be low).
• Your PNH suddenly starts getting worse.
• You have a hard time using your medications as directed.
• You don’t like your current treatment plan.

As you live with PNH, it’s important to regularly discuss your treatment goals with your health care team. Always tell your doctor about any new symptoms, as this may be a sign that your health condition is changing. Your doctor can help you weigh the benefits and risks of different treatment options. Even if you’ve already had these discussions with your doctor, it may help to bring up these issues again — you may be eligible for new therapies or different ones than you were before.

Find Your Team

On myPNHteam, the site for people with paroxysmal nocturnal hemoglobinuria and their loved ones, members come together to ask questions, offer advice, and share their stories with others who understand life with PNH.

Have you talked with your doctor recently about newer options for the treatment of paroxysmal nocturnal hemoglobinuria? Are you considering changing your PNH treatment plan? Share your experience in the comments below, or start a conversation by posting on your Activities page.