If you’re living with paroxysmal nocturnal hemoglobinuria (PNH), is your current treatment plan working well for you? Are the side effects manageable? If you’ve ever hoped for more PNH treatment options, now is a good time to learn about promising therapies being studied.

Researchers are working on new PNH therapies and treatment plans. These updated strategies may work better than older ones, cause fewer side effects, or help improve quality of life. Some may even help people with PNH live longer.

Whether you were just diagnosed or have been managing PNH for years, it’s important to know all your options. Check with your healthcare team if you’re interested in learning more about new treatments and exploring whether a new option may be a better fit for you.

New PNH Drugs

Current PNH treatments such as eculizumab (Soliris) work by blocking your complement system — the part of your immune system that mistakenly attacks red blood cells in people with PNH. Approved by the U.S. Food and Drug Administration (FDA) in 2007, eculizumab blocks a complement protein called factor C5. In recent years, the FDA also approved biosimilars for eculizumab. These are considered interchangeable with Soliris and include eculizumab-aeeb (Bkemv) and eculizumab-aagh (Epysqli).

Newer medications may work differently to block parts of the complement system and help manage PNH symptoms.

Iptacopan for Adults With PNH

Iptacopan (Fabhalta) is an oral medication — the first of its kind for treating adults with PNH — approved by the FDA in 2023. This drug blocks a different complement protein called factor B.

In a study of nearly 100 people with PNH, researchers found that iptacopan helped reverse hemolysis (the breakdown of red blood cells). Most people taking the drug no longer needed blood transfusions.

Iptacopan may also help keep red blood cell levels closer to normal, which could lead to fewer symptoms and a better quality of life. Side effects may include headache, nausea, diarrhea, and rashes.

Pegcetacoplan for Adults With PNH

In 2021, the FDA approved pegcetacoplan (Empaveli) to treat PNH in adults. Pegcetacoplan is a lab-made monoclonal antibody — a type of protein designed to target specific parts of the immune system. This drug is the first PNH treatment to block complement factor C3, a different protein than those targeted by eculizumab and iptacopan.

Studies show that pegcetacoplan works well to reverse PNH. In one clinical trial, researchers tracked hemoglobin levels to measure the drug’s effectiveness. Hemoglobin is a protein inside red blood cells. When these cells break apart, hemoglobin is released into the blood and removed in the urine, which is where the term “hemoglobinuria” comes from.

In a study published in The New England Journal of Medicine, people taking pegcetacoplan had greater increases in hemoglobin levels than those using eculizumab. This suggests the drug helped protect red blood cells from being destroyed.

Importantly, no one in the study developed meningitis, an infection of the membranes covering the spinal cord and brain. This infection has occurred in some people treated with eculizumab, which is why vaccination is recommended with that drug.

In some cases, people with PNH need blood transfusions (receiving donated blood). However, in this study, 85 percent of people who took pegcetacoplan no longer needed transfusions, compared with 15 percent of those who used eculizumab.

Some people with PNH have fewer symptoms after switching from eculizumab to pegcetacoplan. Other studies also showed positive outcomes when participants who had never used PNH medications started taking pegcetacoplan.

Pegcetacoplan is generally safe. Side effects can include diarrhea, and the risk of infection appears similar to eculizumab. Overall, research suggests that pegcetacoplan may be a helpful option for many people with PNH.

Ravulizumab for Adults and Children With PNH

Ravulizumab (Ultomiris) is a complement inhibitor that works similarly to eculizumab — both drugs block complement factor C5. Studies show that the two treatments are about equally safe and effective. However, eculizumab is given every two weeks, whereas ravulizumab is taken every two months.

The FDA approved ravulizumab for adults with PNH in 2018 and for children and teens in 2021. Today, ravulizumab can be used by anyone with PNH who’s over 1 month old. Research shows that ravulizumab may work better than eculizumab to improve the well-being of children with PNH.

Danicopan for Adults With PNH

In 2024, danicopan (Voydeya) became the first drug approved to treat PNH by targeting complement factor D. Danicopan is approved as add-on therapy for people whose PNH symptoms aren’t well controlled by a C5 inhibitor such as eculizumab or ravulizumab alone.

In clinical trials, danicopan was shown to be safe and effective at improving hemoglobin levels and reducing fatigue.

Crovalimab-Akkz for Ages 13 and Up

In 2024, the FDA approved crovalimab-akkz (PiaSky) to treat PNH in people 13 and older who weigh at least 88 pounds. Like eculizumab, crovalimab-akkz is a C5 inhibitor. However, one key difference is how the drug is given. Eculizumab is delivered by IV infusion (into a vein), whereas crovalimab-akkz is given as a subcutaneous injection (under the skin) every four weeks, after initial loading doses.

In a clinical trial comparing the two drugs, crovalimab-akkz worked as well as eculizumab to control hemolysis and reduce the need for blood transfusions.

Bone Marrow Transplantation

PNH affects the bone marrow — the part of your body that makes new blood cells. Genetic mutations (changes) in these cells cause red blood cells to form without protective proteins. This makes them more likely to be attacked and destroyed by the complement system.

An allogeneic bone marrow transplant replaces damaged bone marrow with healthy donor 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. However, this treatment isn’t right for everyone because it carries serious risks, including life-threatening side effects.

Bone marrow transplants have been used for many years to treat PNH, but researchers are still learning how to improve outcomes. Recent studies show that certain factors lead to better results after transplant. You’re more likely to live longer after this procedure if, before getting a transplant, you:

• Are under age 30
• Have needed 20 or fewer blood transfusions
• Have not had active hemolysis

Your doctor may consider these factors when deciding if a bone marrow transplant is a good option for you. You may also be a candidate for transplant if you have not responded well to treatment like eculizumab.

Drugs Being Studied for the Treatment of PNH

New therapies for PNH are also being tested in clinical trials. If the treatments appear to be safe and effective, they may be approved by the FDA and become available to more people living with PNH. Some of these new medications target the same complement proteins as current treatments, while others work in different ways.

Researchers are exploring approaches such as:

• Gene therapy — Uses a virus to deliver healthy copies of the mutated gene directly into blood-forming cells in the bone marrow
• Bifunctional fusion protein — A lab-made protein that works in two ways, blocking C5 and regulating another complement protein called factor H

Talk to Your Doctor About New PNH Treatments

If you’re not satisfied with your current PNH treatment plan, the first step is to talk with your hematology-oncology care team. Your doctor may also suggest switching treatments if they think a different option could work better for you.

It may be time to consider a new treatment if:

• Your current medication isn’t helping as much as expected (for example, if you need frequent blood transfusions or your hemoglobin levels stay low).
• Your PNH symptoms suddenly get worse.
• You have trouble taking your medications as prescribed.
• You’re unhappy with your current treatment plan.

Living with PNH means your needs may change over time, so it’s important to regularly talk with your healthcare team about your treatment goals. Always tell your doctor about any new symptoms, which may be a sign that your 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, they’re worth revisiting. You may be eligible for new therapies or treatments that weren’t available 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.