ASPAVELI: Uses, Dosage & Side Effects
A complement C3 inhibitor for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults with anemia, targeting both intravascular and extravascular hemolysis
ASPAVELI (pegcetacoplan) is a first-in-class complement C3 inhibitor used for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults who have anemia despite or independent of prior complement inhibitor therapy. PNH is a rare, life-threatening blood disorder in which the complement system – part of the body’s immune defense – mistakenly attacks and destroys red blood cells. By binding to complement protein C3, pegcetacoplan blocks both the intravascular (within blood vessels) and extravascular (in the liver and spleen) pathways of red blood cell destruction. ASPAVELI is administered as a subcutaneous infusion and has demonstrated significant improvements in hemoglobin levels and reduced transfusion requirements in clinical trials. It requires a prescription and is initiated under specialist supervision.
Quick Facts: ASPAVELI
Key Takeaways
- ASPAVELI (pegcetacoplan) is the first complement C3 inhibitor approved for PNH, addressing both intravascular and extravascular hemolysis – a limitation of earlier C5-targeted therapies like eculizumab and ravulizumab.
- Vaccination against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae is mandatory at least 2 weeks before starting treatment, as complement inhibition increases the risk of serious infections with encapsulated bacteria.
- The standard dose is 1,080 mg administered subcutaneously twice weekly; after initial supervised doses, patients may self-administer at home using an infusion pump or on-body delivery system.
- In the pivotal PEGASUS trial, pegcetacoplan demonstrated superiority over eculizumab in improving hemoglobin levels and reducing the need for blood transfusions in PNH patients with persistent anemia.
- Abrupt discontinuation should be avoided, as it may trigger rebound hemolysis; patients must be monitored for at least 8 weeks after stopping treatment for signs of complement-mediated red blood cell destruction.
What Is ASPAVELI and What Is It Used For?
ASPAVELI contains the active substance pegcetacoplan, a pegylated cyclic peptide that represents a significant advancement in the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Pegcetacoplan was specifically designed to bind to complement protein C3 and its activation fragment C3b, which are central components of the complement system – a key part of the body’s innate immune defense. By targeting C3, pegcetacoplan inhibits the complement cascade at a level upstream of C5, thereby addressing a broader range of complement-mediated damage to red blood cells than previous treatments that targeted only C5.
The complement system is a complex network of proteins that circulates in the blood and plays a crucial role in the body’s defense against infections. Under normal conditions, healthy red blood cells are protected from complement attack by surface proteins, particularly CD55 (also known as decay-accelerating factor, DAF) and CD59 (membrane inhibitor of reactive lysis, MIRL). In PNH, a somatic mutation in the PIGA gene in hematopoietic stem cells leads to a deficiency of glycosylphosphatidylinositol (GPI)-anchored proteins, including CD55 and CD59. Without these protective surface proteins, red blood cells become vulnerable to complement-mediated destruction.
The destruction of red blood cells in PNH occurs through two main pathways. Intravascular hemolysis is caused by the formation of the membrane attack complex (MAC, C5b-9), which punches holes in the red blood cell membrane, causing the cell to rupture directly in the bloodstream. This releases free hemoglobin into the plasma, leading to dark-colored urine (hemoglobinuria), fatigue, abdominal pain, difficulty swallowing, erectile dysfunction, and an increased risk of blood clots. Extravascular hemolysis occurs when C3b fragments are deposited on the surface of red blood cells (opsonization), marking them for destruction by macrophages in the liver and spleen. This pathway contributes to ongoing anemia even in patients receiving C5-inhibitor therapy, as C5 inhibitors do not prevent C3b opsonization.
By binding to and blocking C3, pegcetacoplan prevents the complement system from attacking red blood cells through both pathways. This dual mechanism of action is a key advantage over C5 inhibitors (such as eculizumab and ravulizumab), which block only intravascular hemolysis mediated by the MAC complex but do not address C3b-mediated extravascular hemolysis. Clinical evidence demonstrates that ASPAVELI can increase hemoglobin levels, reduce the need for blood transfusions, and improve quality of life in PNH patients, including those who had persistent anemia despite treatment with a C5 inhibitor.
ASPAVELI is approved by the European Medicines Agency (EMA) and marketed in the EU for the treatment of adult patients with PNH who have anemia. In the United States, pegcetacoplan is marketed under the brand name EMPAVELI and has been approved by the FDA for PNH in adults since 2021. The drug is intended for long-term use, as PNH is a chronic, lifelong condition.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired blood disorder affecting approximately 1–2 per million people worldwide. Despite its name suggesting symptoms occur mainly at night, hemolysis is continuous. The “paroxysmal” nature refers to episodes of worsened hemolysis that can be triggered by infections, surgery, stress, or other complement-activating events. PNH can cause severe anemia, blood clots (thrombosis), kidney damage, and significant impairment of quality of life. Without treatment, PNH can be life-threatening, particularly due to thromboembolic complications.
What Should You Know Before Using ASPAVELI?
Contraindications
There are specific situations in which ASPAVELI must not be used. Understanding these absolute contraindications is essential before initiating treatment.
- Hypersensitivity: Do not use ASPAVELI if you are allergic to pegcetacoplan or any of the other ingredients in this medicine, including sorbitol, acetic acid, sodium acetate trihydrate, sodium hydroxide, and water for injections.
- Active infection with encapsulated bacteria: ASPAVELI must not be used if you currently have an infection caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae, or Haemophilus influenzae. These infections can be particularly severe when the complement system is inhibited.
- Unvaccinated patients: You must have been vaccinated against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae before starting ASPAVELI. Treatment cannot begin if these vaccinations have not been administered.
Warnings and Precautions
ASPAVELI inhibits the complement system, which is part of the body’s defense against infections. This significantly increases the risk of life-threatening infections caused by encapsulated bacteria, including meningococcal disease (Neisseria meningitidis), pneumococcal disease (Streptococcus pneumoniae), and Haemophilus influenzae infections. Even with vaccination, these infections can still occur. Seek immediate medical attention if you develop fever, headache with stiff neck, rash, confusion, severe chills, or other signs of infection.
Before starting ASPAVELI, inform your doctor about any current infections or recent history of infections. Because the complement system is a critical part of immune defense, suppressing it with pegcetacoplan increases susceptibility to a range of infections, particularly those caused by encapsulated organisms.
Your doctor will ensure you receive vaccinations against meningococcal, pneumococcal, and Haemophilus influenzae bacteria at least 2 weeks before your first dose. If vaccination cannot be completed 2 weeks in advance, your doctor will prescribe prophylactic antibiotics for 2 weeks after vaccination to provide interim protection. Even after vaccination, you should be aware that vaccines reduce but do not completely eliminate the risk of these serious infections. Your doctor may also consider additional preventive measures such as prophylactic antibiotics, in accordance with national guidelines.
Seek immediate medical attention if you experience any of the following symptoms, which may indicate a serious infection:
- Headache with fever
- Fever with or without rash
- Fever with shaking chills
- Shortness of breath or rapid heart rate
- Cold, clammy skin
- Headache with stiff neck or stiff back
- Headache with nausea or vomiting
- Sensitivity to light
- Muscle pain with flu-like symptoms
- Confusion or extreme pain or discomfort
Allergic Reactions
Some patients may experience allergic reactions to ASPAVELI. If a serious allergic reaction occurs during infusion, stop the infusion immediately and seek urgent medical attention. Signs of a severe allergic reaction (anaphylaxis) can include difficulty breathing, chest pain or tightness, dizziness or feeling faint, severe itching or raised bumps on the skin (hives), and swelling of the face, lips, tongue, or throat that may cause difficulty swallowing.
Injection Site Reactions
Reactions at the site of subcutaneous infusion have been commonly reported with ASPAVELI. These include redness (erythema), swelling, itching (pruritus), bruising, and pain. Most injection site reactions are mild to moderate and resolve within a few days. Proper training in injection technique is important to minimize these reactions. Rotating injection sites between administrations also helps reduce local irritation.
Laboratory Monitoring
During treatment with ASPAVELI, your doctor will perform regular blood tests to monitor lactate dehydrogenase (LDH) levels, hemoglobin, reticulocyte counts, and kidney function. These parameters help assess the effectiveness of treatment and detect any complications. Your doctor may adjust the dose based on these results. Additionally, the use of silica-based reagents in coagulation tests should be avoided, as pegcetacoplan can cause artificially prolonged activated partial thromboplastin time (aPTT) results when such reagents are used.
Pregnancy and Breastfeeding
The effects of ASPAVELI on an unborn child are not known. As a precautionary measure, ASPAVELI is not recommended during pregnancy. Women of childbearing potential should use effective contraception during treatment and for up to 8 weeks after the last dose. If you are pregnant, think you may be pregnant, or are planning to become pregnant, consult your doctor before using this medicine.
It is not known whether pegcetacoplan or its metabolites pass into human breast milk. ASPAVELI is not recommended during breastfeeding. If you are breastfeeding, discuss with your doctor whether you should continue breastfeeding during treatment.
Driving and Operating Machinery
ASPAVELI has no or negligible influence on the ability to drive and use machines. You should not experience any impairment of these activities from the medication itself.
Important Information About Ingredients
Sorbitol: ASPAVELI contains sorbitol (E420), which is a source of fructose. If you have been told by your doctor that you have an intolerance to some sugars, or if you have been diagnosed with hereditary fructose intolerance (HFI), a rare genetic condition in which the body cannot break down fructose, talk to your doctor before using this medicine.
Sodium: This medicine contains less than 1 mmol sodium (23 mg) per dose, meaning it is essentially “sodium-free.”
How Does ASPAVELI Interact with Other Drugs?
Drug interaction studies with pegcetacoplan are limited. However, because ASPAVELI works by inhibiting the complement system rather than through hepatic enzyme metabolism (like CYP450 enzymes), the potential for pharmacokinetic interactions with most conventional drugs is considered low. Nevertheless, it is important to inform your doctor about all medications, supplements, and herbal products you are taking, as interactions involving the immune system can have clinically significant consequences.
Major Interactions
| Interacting Drug | Effect | Clinical Significance |
|---|---|---|
| C5 inhibitors (eculizumab, ravulizumab) | Overlap period required during switch; abrupt C5 inhibitor discontinuation without overlap can cause severe hemolysis rebound | Must co-administer for 4 weeks during transition, then stop C5 inhibitor |
| Live vaccines (e.g., MMR, varicella, BCG, live influenza) | Risk of vaccine-strain infection due to complement inhibition and potential immunosuppression | Avoid live vaccines during treatment; use inactivated alternatives |
| Silica-based coagulation reagents | Artificially prolonged aPTT results in laboratory tests | Use non-silica reagents for aPTT testing; clinical coagulation is unaffected |
Minor Interactions
| Interacting Drug | Effect | Clinical Significance |
|---|---|---|
| Immunosuppressive agents (e.g., corticosteroids, azathioprine) | Potential additive immunosuppression, increased infection risk | Monitor closely for signs of infection; consider prophylactic measures |
| Anticoagulants (e.g., warfarin, heparin) | No direct pharmacological interaction, but PNH itself increases thrombotic risk | Continue anticoagulation as prescribed; monitor coagulation parameters |
| Erythropoiesis-stimulating agents (e.g., erythropoietin) | May be used concurrently to support red blood cell production | No negative interaction; commonly co-administered in PNH management |
| Iron and folic acid supplements | May be needed to support red blood cell production during recovery from anemia | No interaction; often recommended as supportive care |
Because ASPAVELI is a biologic peptide that acts on the complement system rather than being metabolized by liver enzymes, it has a low potential for traditional drug–drug interactions. However, the immunological consequences of complement inhibition mean that concomitant use with other immunosuppressive therapies should be carefully evaluated. Your doctor will assess the overall risk–benefit ratio of any combination therapy.
What Is the Correct Dosage of ASPAVELI?
ASPAVELI is always administered as a subcutaneous (under the skin) infusion. The first doses are given by a healthcare professional in a clinical setting to ensure safety and proper technique. If treatment goes well, your doctor may discuss the possibility of self-administration at home after you or a caregiver have been trained in the correct infusion technique.
Adults
Standard Dosing
Dose: 1,080 mg subcutaneously twice per week (days 1 and 4 of each treatment week)
Concentration: 54 mg/mL in a 20 mL single-use vial
Duration: Long-term (PNH is a chronic condition; treatment is expected to continue indefinitely)
Do not change the dose or dosing interval without consulting your doctor. Your doctor may adjust the dosing schedule to 1,080 mg every third day (e.g., days 1, 4, 7, 10, 13, etc.) if clinically appropriate based on your response and laboratory parameters.
Switching from a C5 Inhibitor
Overlap period: If you are switching to ASPAVELI from a C5 inhibitor (eculizumab or ravulizumab), you should take ASPAVELI in addition to your current C5 inhibitor dose for 4 weeks.
After 4 weeks: Discontinue the C5 inhibitor and continue ASPAVELI alone.
This overlap period is critical to prevent a rebound of hemolysis during the transition. Your doctor will monitor your blood parameters closely during this period.
Administration Methods
ASPAVELI can be administered using one of two methods:
- Infusion pump with syringe system: The infusion takes approximately 30 minutes when using 2 infusion sites, or approximately 60 minutes when using 1 infusion site.
- On-body delivery system (OBDS): The infusion time is typically 30–60 minutes, depending on how quickly the medicine flows into the body.
Suitable infusion sites include the abdomen (avoiding the area within 5 cm of the navel), thighs, hips, and upper arms. You should rotate injection sites between administrations, ensuring at least 7.5 cm between multiple infusion sites. Avoid areas where the skin is tender, bruised, red, or hard, and avoid tattooed, scarred, or stretch-marked skin.
The infusion must be started immediately after drawing the solution into the syringe and completed within 2 hours of syringe preparation. The solution should be clear, colorless to slightly yellowish. Do not use if the solution is cloudy, contains particles, or has changed color.
Children
ASPAVELI is intended for use in adult patients only. The safety and efficacy of pegcetacoplan in children and adolescents under 18 years of age have not been established, and there are no available data in this age group. It is not recommended for use in pediatric patients.
Missed Dose
If you miss a dose, take it as soon as possible. Then take your next dose at the usual scheduled time. Do not take a double dose to make up for a missed one. If you are unsure about what to do, contact your doctor as soon as possible.
Stopping Treatment
PNH is a lifelong condition and you are expected to use ASPAVELI long term. If you wish to stop treatment, talk to your doctor first. Abruptly stopping ASPAVELI can lead to a rebound of complement-mediated hemolysis, with potentially serious consequences including severe anemia, blood clots, dark urine, abdominal pain, shortness of breath, fatigue, and difficulty swallowing. Your doctor will monitor you closely for at least 8 weeks after stopping treatment.
Signs and symptoms that may occur after discontinuation due to rebound hemolysis include:
- Fatigue and weakness
- Shortness of breath
- Blood in the urine (dark or cola-colored urine)
- Abdominal pain
- Decreased red blood cell count
- Blood clots (thrombosis)
- Difficulty swallowing
- Erectile dysfunction in men
Contact your doctor immediately if you experience any of these signs or symptoms after stopping ASPAVELI.
What Are the Side Effects of ASPAVELI?
Like all medicines, ASPAVELI can cause side effects, although not everyone gets them. Your doctor will discuss the potential side effects with you and explain the risks and benefits of treatment before you begin. The most serious risk associated with ASPAVELI is severe infection, particularly with encapsulated bacteria (see section on Warnings and Precautions). If you experience any symptoms of infection, seek immediate medical attention.
Very Common
May affect more than 1 in 10 people
- Injection site reactions (redness, swelling, itching, bruising, pain) – usually resolve within a few days
- Upper respiratory tract infections (nose, throat, or airways)
- Diarrhea
- Hemolysis (breakdown of red blood cells)
- Abdominal pain (stomach pain)
- Headache
- Fatigue (tiredness)
- Fever (pyrexia)
- Cough
- Urinary tract infection
- Vaccination-related complications
- Pain in arms and legs (extremity pain)
- Dizziness
- Joint pain (arthralgia)
- Back pain
Common
May affect up to 1 in 10 people
- Injection site reaction with redness or skin hardening (induration)
- Infections of the ear, mouth, or skin
- Sore throat
- Decreased platelets (thrombocytopenia) – may lead to bruising or bleeding more easily
- Nausea
- Low potassium levels in the blood (hypokalemia)
- Nosebleed (epistaxis)
- Skin redness (erythema)
- Muscle pain (myalgia)
- Gastrointestinal infection (stomach and bowel infection)
- Elevated liver enzyme levels
- Shortness of breath (dyspnea)
- Decreased white blood cells (neutropenia)
- Decreased kidney function
- Changes in urine color
- High blood pressure (hypertension)
- Muscle spasms
- Nasal congestion
- Skin rash
- Bloodstream infection (sepsis)
- Viral infection
- Fungal infection
- Respiratory tract infection
- Eye infection
- Hives (urticaria)
- COVID-19
- Bacterial infection
- Vaginal infection
Uncommon
May affect up to 1 in 100 people
- Cervicitis (inflammation of the cervix)
- Groin infection
- Nasal abscess (pocket of pus in the nose)
- Pneumonia (lung infection)
- Tuberculosis
- Esophageal yeast infection (candidiasis)
- Anal abscess (perianal abscess)
Reporting Side Effects
If you experience any side effects, including those not listed above, tell your doctor, pharmacist, or nurse. You can also report suspected side effects to your national pharmacovigilance authority (e.g., the EMA in Europe, the FDA MedWatch program in the United States, or the MHRA Yellow Card Scheme in the United Kingdom) to help continuously monitor the benefit–risk balance of ASPAVELI.
How Should ASPAVELI Be Stored?
Keep this medicine out of the sight and reach of children. Do not use after the expiry date (EXP) printed on the carton and vial label. The expiry date refers to the last day of the indicated month.
- Storage temperature: Store in a refrigerator at 2°C to 8°C (36°F to 46°F). Do not freeze.
- Light protection: Keep the vial in the outer carton to protect from light.
- Before use: Remove one vial from the refrigerator and allow it to warm to room temperature for approximately 30 minutes. Do not use a microwave or any other heat source to speed up warming.
- After preparation: Once drawn up into the syringe, the infusion must be started immediately and completed within 2 hours.
- Inspection: ASPAVELI is a clear, colorless to slightly yellowish solution. Do not use if the solution is cloudy, contains particles, or has a dark yellow color. Also check that the protective flip-off cap is intact and the expiry date has not passed.
Do not dispose of medicines via household waste or wastewater. Ask your pharmacist how to properly dispose of medicines that are no longer needed. These measures help protect the environment.
What Does ASPAVELI Contain?
Active Substance
The active substance is pegcetacoplan, a pegylated cyclic peptide that binds to complement protein C3 and its activation fragment C3b. Each single-use vial contains 1,080 mg of pegcetacoplan at a concentration of 54 mg/mL in 20 mL of solution. Pegcetacoplan is produced through chemical synthesis and pegylation, which extends the drug’s half-life in the body, allowing twice-weekly subcutaneous administration.
Inactive Ingredients (Excipients)
- Sorbitol (E420) – see section on Important Information About Ingredients regarding fructose intolerance
- Glacial acetic acid (concentrated)
- Sodium acetate trihydrate
- Sodium hydroxide (for pH adjustment)
- Water for injections
Appearance and Pack Sizes
ASPAVELI is a clear, colorless to slightly yellowish aqueous solution for subcutaneous infusion supplied in a 20 mL single-use glass vial. Solutions that are cloudy, contain visible particles, or have changed color must not be used.
ASPAVELI is available in a single-vial pack or as a multipack containing 1 × 8 vials. Alcohol swabs, needles, and other infusion accessories or equipment are not included in the package and must be obtained separately. Not all pack sizes may be marketed in every country.
Marketing Authorization Holder and Manufacturer
The marketing authorization holder and manufacturer is Swedish Orphan Biovitrum AB (publ), commonly known as Sobi. Additional information about ASPAVELI is available on the European Medicines Agency website. In the United States, pegcetacoplan is marketed by Apellis Pharmaceuticals under the brand name EMPAVELI.
Frequently Asked Questions About ASPAVELI
ASPAVELI (pegcetacoplan) is used to treat adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have anemia due to the disease. PNH is a rare blood disorder where the immune system’s complement cascade attacks red blood cells, causing their destruction (hemolysis). ASPAVELI binds to complement protein C3, blocking both intravascular and extravascular hemolysis, which can improve hemoglobin levels and reduce the need for blood transfusions.
Eculizumab and ravulizumab are C5 inhibitors that block the terminal complement pathway and prevent intravascular hemolysis. However, they do not prevent C3b deposition on red blood cells, which can lead to ongoing extravascular hemolysis in the liver and spleen. ASPAVELI targets complement C3, which is upstream of C5, thereby blocking both intravascular and extravascular hemolysis. This makes it particularly useful for patients who continue to have anemia despite C5 inhibitor therapy. Additionally, ASPAVELI is administered subcutaneously (self-injectable at home), while eculizumab requires intravenous infusion in a clinical setting.
Yes, after your first doses are administered by healthcare professionals in a clinical setting, your doctor may determine that home self-administration is appropriate. You or a caregiver will receive thorough training on how to prepare and administer the subcutaneous infusion using an infusion pump or on-body delivery system. Proper injection technique, site rotation, and recognition of potential complications will be covered. Only begin self-injecting once your healthcare team confirms you are competent to do so.
ASPAVELI works by inhibiting complement protein C3, which is a critical component of the body’s immune defense against certain bacteria. When C3 is blocked, the body becomes much more vulnerable to infections caused by encapsulated bacteria, particularly Neisseria meningitidis (meningococcal disease), Streptococcus pneumoniae (pneumococcal disease), and Haemophilus influenzae. These infections can be life-threatening. Vaccination provides your immune system with specific antibodies against these bacteria, significantly reducing (though not eliminating) the risk of severe infection during treatment.
Abruptly stopping ASPAVELI can lead to a rebound of complement-mediated hemolysis, meaning the complement system can rapidly resume destroying your red blood cells. This can cause severe symptoms including worsening anemia, dark urine, fatigue, abdominal pain, shortness of breath, blood clots, difficulty swallowing, and erectile dysfunction. If your doctor decides to discontinue treatment, you will be monitored closely for at least 8 weeks for signs of hemolysis. Never stop treatment on your own without first consulting your doctor.
The effects of ASPAVELI on an unborn child are not known, as there are insufficient data from its use in pregnant women. As a precautionary measure, ASPAVELI is not recommended during pregnancy or breastfeeding. Women of childbearing potential should use effective contraception during treatment and for up to 8 weeks after the last dose. If you become pregnant during treatment or are planning a pregnancy, discuss your options with your hematologist, who can weigh the risks and benefits of continuing or modifying therapy.
References
- European Medicines Agency (EMA). ASPAVELI (pegcetacoplan) – Summary of Product Characteristics. Last updated 2025. Available from: EMA EPAR.
- U.S. Food and Drug Administration (FDA). EMPAVELI (pegcetacoplan) Prescribing Information. Revised 2024. Available from: FDA Drug Label.
- Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal haemoglobinuria (PEGASUS): a randomised, open-label, phase 3 trial. Lancet. 2021;397(10286):1728–1740. doi:10.1016/S0140-6736(21)00857-5.
- de Latour RP, Szer J, Scheinberg P, et al. Pegcetacoplan versus eculizumab in PNH (PEGASUS): 48-week follow-up from a randomised, open-label, phase 3 trial. Lancet Haematol. 2022;9(9):e697–e709.
- Peffault de Latour R, Brodsky RA, Ortiz S, et al. Pharmacokinetic and pharmacodynamic effects of pegcetacoplan in patients with PNH. Blood Adv. 2023;7(3):381–390.
- Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124(18):2804–2811. doi:10.1182/blood-2014-02-522128.
- Risitano AM, Marotta S, Ricci P, et al. Anti-complement treatment for paroxysmal nocturnal hemoglobinuria: time for proximal complement inhibition? A position paper from the SAAWP of the EBMT. Front Immunol. 2019;10:1157.
- Hill A, DeZern AE, Kinoshita T, Brodsky RA. Paroxysmal nocturnal haemoglobinuria. Nat Rev Dis Primers. 2017;3:17028. doi:10.1038/nrdp.2017.28.
- World Health Organization (WHO). WHO Model List of Essential Medicines – 24th List. Geneva: WHO; 2025.
- Wong RSM, Pullon HWH, Amine I, et al. Pegcetacoplan in PNH: long-term follow-up from the phase 3 PEGASUS and PRINCE trials. Blood. 2023;142(Suppl 1):3527.
Editorial Team
This article was written and reviewed by the iMedic Medical Editorial Team, comprising licensed specialist physicians with expertise in hematology, immunology, and clinical pharmacology.
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