PNH (Paroxysmal Nocturnal Hemoglobinuria) Treatments Compared
6 FDA-approved drugs, side by side from the current prescribing labels. Newest label Aug 2026. Checked October 23, 2026.
The full Paroxysmal Nocturnal Hemoglobinuria guide, with open trials and help paying →
Six medicines are FDA approved for paroxysmal nocturnal hemoglobinuria (PNH), and they block the complement system at 2 different points. Soliris, Ultomiris and Piasky block C5, the last step, which stops red blood cells from bursting inside blood vessels; Bkemv and Epysqli are biosimilar versions of Soliris. Empaveli, Fabhalta and Voydeya act earlier in the chain and also target red cells cleared in the spleen and liver; Voydeya is only added to Ultomiris or Soliris. Dosing ranges from clinic visits every 2, 4 or 8 weeks to a twice-weekly home infusion or pills 2 or 3 times a day. All 6 carry a boxed warning for serious infections, need vaccines before the first dose and are available only through a REMS safety program.
6 approved PNH treatments at a glance
How each drug is given, how often, who it is approved for and when it was first approved, from the current FDA label.
A year on each PNH treatment
Every dose in a year, drawn to the label schedule, so you can see at a glance how often each one is given.
Counts follow the label schedule for a full year on the maintenance dose. Drugs given in cycles or adjusted to blood tests have no fixed count, so their schedule is written out instead.
What the pivotal PNH trials showed
The primary result from the trial each FDA approval rests on, as reported on the label.
Paroxysmal Nocturnal Hemoglobinuria treatment side effects and warnings
Soliris, Ultomiris, Piasky, Empaveli, Fabhalta and Voydeya carry a boxed warning, the FDA's strongest. The rest list their main precautions. Side effect rates show how many people in each drug's trials reported each one, whether or not the drug caused it.
Help paying for PNH treatment
Drugmaker copay and free-drug programs, plus charity funds for the condition, checked every week.
One email when the FDA acts on a Paroxysmal Nocturnal Hemoglobinuria treatment, a label changes or a new trial opens. Never a newsletter.
You'll get new recruiting trials, changes to trials, FDA decisions and condition news for Paroxysmal Nocturnal Hemoglobinuria. Turn any of these off from the link in every email.
Paroxysmal Nocturnal Hemoglobinuria treatment questions people ask
Soliris vs Ultomiris for PNH: what is the difference?
Both block complement protein C5 and both come from Alexion, part of AstraZeneca; Ultomiris is the longer-acting version.
Who it is for: Ultomiris is approved for adults and children 1 month and older with PNH. The Soliris label gives PNH dosing only for adults.
How it is given: Soliris is an IV infusion every 2 weeks after 4 weekly starting doses and a fifth dose at week 5. Ultomiris is an IV infusion every 8 weeks for people who weigh 20 kg or more, after a loading dose and a first maintenance dose 2 weeks later.
Trial results: In people new to treatment, 73.6% on Ultomiris and 66.1% on Soliris avoided transfusions over 26 weeks. The trial was built to show Ultomiris was no worse than Soliris, not that it was better.
Safety: Both carry the same boxed warning for serious meningococcal infections, need meningococcal vaccines first and share 1 REMS safety program.
Source: FDA label for Soliris, DailyMed, effective July 2026; FDA label for Ultomiris, DailyMed, effective October 2025
Empaveli vs Fabhalta for PNH: what is the difference?
Both act early in the complement chain and are used on their own, Empaveli at C3 and Fabhalta at factor B. Both are approved for adults.
How it is given: Empaveli is an infusion under the skin twice a week, given at home with a small pump or a wearable injector. Fabhalta is a capsule taken by mouth twice a day.
Trial results: Both main trials enrolled adults who were still anemic on a C5 inhibitor. On Empaveli, hemoglobin rose 2.37 g/dL at week 16 while it fell 1.47 g/dL in people who stayed on Soliris. On Fabhalta, 82.3% raised hemoglobin by at least 2 g/dL without transfusions at 24 weeks, against 0% of people who stayed on Soliris or Ultomiris. The trials measured different things, so the numbers do not rank the 2 drugs.
Safety: Both carry a boxed warning for serious infections from encapsulated bacteria and require vaccines and a REMS program. After stopping, the labels call for watching for hemolysis for at least 8 weeks on Empaveli and 2 weeks on Fabhalta.
More: Empaveli drug page and Fabhalta drug page
Source: FDA label for Empaveli, DailyMed, effective August 2026; FDA label for Fabhalta, DailyMed, effective July 2026
What is Voydeya and who is it for?
Voydeya (danicopan) is a tablet taken 3 times a day on top of Ultomiris or Soliris. It is approved for adults with PNH who have extravascular hemolysis. That means red cells are still being cleared in the spleen and liver, even though the C5 inhibitor stops them bursting in the blood vessels.
Not on its own: The label says Voydeya has not been shown to work alone and should only be added to Ultomiris or Soliris.
Trial results: In ALPHA, adding Voydeya raised hemoglobin 2.9 g/dL at week 12, against 0.5 g/dL with a placebo add-on. Transfusions were avoided by 83.3% against 38.1%.
Watch-outs: A boxed warning for serious infections from encapsulated bacteria, vaccines and a REMS program, plus checks for liver enzymes and cholesterol.
More: Voydeya drug page
Which PNH treatments can be taken at home?
Fabhalta is a capsule twice a day, and Voydeya is a tablet 3 times a day added to a C5 inhibitor. Empaveli is an infusion under the skin twice a week that patients or caregivers can give at home after training.
Given by a healthcare provider: Piasky is an injection under the skin every 4 weeks, and its label says only healthcare providers should give it. Soliris and Ultomiris are IV infusions.
More: Fabhalta drug page, Empaveli drug page and Voydeya drug page
Source: FDA label for Fabhalta, DailyMed, effective July 2026; FDA label for Empaveli, DailyMed, effective August 2026; FDA label for Voydeya, DailyMed, effective May 2026; FDA label for Piasky, DailyMed, effective November 2025
How is Piasky different from Soliris and Ultomiris?
Piasky (crovalimab) also blocks C5, but it is an injection under the skin every 4 weeks after a first IV dose and 4 weekly starting injections.
Who it is for: People 13 and older with PNH who weigh at least 40 kg.
Trial results: In COMMODORE 2, 65.7% of people new to treatment on Piasky and 68.1% on Soliris avoided transfusions over 24 weeks. That met the goal of being no worse than Soliris.
Switching: Piasky binds a different spot on C5. In trials, 19% of people who switched from Soliris or Ultomiris had Type III hypersensitivity reactions, such as joint pain, rash or fever. The label calls for watching for these for 30 days after a switch.
More: Piasky drug page
Source: FDA label for Piasky, DailyMed, effective November 2025
Is there a biosimilar for Soliris?
Yes, 2. Bkemv (eculizumab-aeeb) from Amgen was approved May 28, 2024, and Epysqli (eculizumab-aagh) from Samsung Bioepis, sold in the US by Teva, was approved July 19, 2024.
Same dosing: Both labels give the same PNH dosing as Soliris and rely on the same PNH trial.
Same warning: Both carry the boxed warning for serious meningococcal infections, and each has its own REMS program.
Availability: Teva announced Epysqli available in the US on April 7, 2025. Amgen told the Vermont Attorney General that Bkemv became commercially available on April 2, 2025.
Ultomiris: The FDA label database lists no biosimilar of Ultomiris as of October 2026.
Source: FDA label for Bkemv, DailyMed, effective July 2026; FDA label for Epysqli, DailyMed, effective July 2026; Teva and Samsung Bioepis, April 7, 2025
What new PNH treatments are coming?
We found no PNH drug with a public FDA decision date as of October 23, 2026.
Pozelimab plus cemdisiran: Regeneron's 2 drugs, given together under the skin every 4 weeks, block C5 in 2 ways. In an early group of the ACCESS-1 trial, 96% on the combination and 80% on Ultomiris kept LDH controlled on average from weeks 8 to 26, according to Regeneron. Regeneron expects results from the larger group, tested against Soliris, in late 2026.
Zaltenibart: An antibody against MASP-3 given by IV every 8 weeks. Novo Nordisk bought the rights from Omeros in 2025 after Omeros paused its Phase 3 plans, and Novo said it aims to start a global Phase 3 program.
KP104: Kira Pharmaceuticals reported that all 18 people in its Phase 2 trial raised hemoglobin by at least 2 g/dL, and says Phase 3 trials are being planned.
Source: Regeneron, December 7, 2024; ACCESS-1 on ClinicalTrials.gov; Fierce Biotech, October 15, 2025; Kira Pharmaceuticals, December 10, 2024
More on Paroxysmal Nocturnal Hemoglobinuria at Trial Friend
Where every number on this page comes from
Approved-drug facts are from the current FDA prescribing information on DailyMed, with the label's effective date. Investigational-drug facts are from ClinicalTrials.gov and the sources named in each row.
- Soliris (eculizumab (biosimilars: Bkemv, eculizumab-aeeb; Epysqli, eculizumab-aagh)) prescribing information, Alexion (AstraZeneca); Bkemv from Amgen; Epysqli from Samsung Bioepis, sold in the US by Teva, label dated Jul 2026. DailyMed. Pivotal trial: ClinicalTrials.gov
- Ultomiris (ravulizumab-cwvz) prescribing information, Alexion (AstraZeneca), label dated Oct 2025. DailyMed. Pivotal trial: ClinicalTrials.gov
- Piasky (crovalimab-akkz) prescribing information, Genentech (Roche); discovered by Chugai, label dated Nov 2025. DailyMed. Pivotal trial: ClinicalTrials.gov
- Empaveli (pegcetacoplan) prescribing information, Apellis Pharmaceuticals, label dated Aug 2026. DailyMed. Pivotal trial: ClinicalTrials.gov
- Fabhalta (iptacopan) prescribing information, Novartis, label dated Jul 2026. DailyMed. Pivotal trial: ClinicalTrials.gov
- Voydeya (danicopan) prescribing information, Alexion (AstraZeneca), label dated May 2026. DailyMed. Pivotal trial: ClinicalTrials.gov
This page compares what the FDA labels and pivotal trials report. It is not medical advice, and no trial on this page compared these drugs with each other. Written and checked by Jason Hunter; the method is on the editorial standards page.