EPYSQLI Eculizumab-aagh 300 mg/30mL Injection, Solution
🆔 Identity & classification
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🏷️ RxNorm drug class
This medicine belongs to the Complement Inhibitor class.
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🏭 Manufacturer & labeler
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🩺 Clinical
Eculizumab injection is used to treat paroxysmal nocturnal hemoglobinuria (PNH: a type of anemia in which too many red blood cells are broken down in the body, so there are not enough healthy cells to bring oxygen to all parts of the body), atypical hemolytic uremic syndrome (aHUS; an inherited condition in which small blood clots form in the body and may cause damage to the blood vessels, blood cells, kidneys, and other parts of the body), a certain type of myasthenia gravis (MG; a disorder of the nervous system that causes muscle weakness), and a certain type of neuromyelitis optica spectru...
Read the full MedlinePlus article ↗- Eculizumab works by turning off part of your immune system — specifically, the complement system. That's what makes it effective, but it also leaves you less able to fight off cert...
- Why do I need to get vaccinated before starting this medication?
- Don't skip or delay doses without talking to your doctor first — this is really important. If you stop getting infusions, even briefly, your underlying condition can come back fast...
- What should I do if I miss an infusion appointment?
Patient education
Supplement & herbal interactions
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Ask a licensed pharmacist directly — free, answered by our team.
🧪 Inactive Ingredients / Excipients
Inactive ingredients, also called excipients, are components of the drug product other than the active ingredient. They may include fillers, dyes, coatings, preservatives, flavors, or other formulation ingredients.
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UNII 6OZP39ZG8H
Polysorbate 80 is a synthetic emulsifier derived from sorbitol and oleic acid. It helps mix oil and water-based ingredients together in medications and improves how the product disperses in the body.
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UNII 70WT22SF4B
A mineral salt that acts as a buffer to maintain the pH balance of the medication. It helps stabilize the drug's potency and prevents unwanted chemical changes during storage.
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UNII 593YOG76RN
A salt made from sodium and phosphate that helps regulate acidity and maintain proper pH balance in the medicine. It also acts as a buffer to keep the product stable.
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UNII 7YIN7J07X4
A natural sugar derived from plants and microorganisms. It acts as a filler to give the medication bulk and stability, and helps preserve the active ingredient during storage and manufacturing.
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UNII 059QF0KO0R
Water is a liquid solvent that dissolves and mixes ingredients together in liquid medicines, syrups, and injections. It helps distribute the active drug evenly throughout the product.
5 inactive ingredients listed in the exact product block matched to this NDC.
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ingredient classCode="IACT" elements from the exact product block matched by this NDC. Label-section narrative from DailyMed / the openFDA label index is shown separately when available.Inactive ingredient FAQ
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💲 Pricing
A drug doesn't have one price. Each row is a different public payment system, and none is what you'd pay at the counter — that depends on your insurance. The ⓘ on each row explains what it measures.
| Price system | Per mL | Per package |
|---|---|---|
| Retail pharmacies payNADAC · weekly | Not in the retail survey — common for institutional, discontinued, or low-volume packs. | |
| Medicaid paysCMS SDUD · 12 mo | No recent Medicaid claims on file for this NDC — rare and low-volume NDCs are suppressed in the public data. | |
| Medicare drug plans payPart D · quarterly | No Part D plan price is available for this NDC in our data. | |
| Medicare Part B allowsASP · Q5151 | $31.767 / Q5151 unit | — |
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🧾 Billing & reimbursement
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🔁 Therapeutic equivalents
| Product | Labeler | Pack | NADAC/unit | TE | Status | Price vs. this |
|---|---|---|---|---|---|---|
| Epysqli 300 mg/30mLthis 51759-0208-13 | Teva | 1 vial | — | — | FDA listed | — |
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⏳ Availability & biosimilar status
1 biosimilar and 1 interchangeable are FDA-licensed for this reference biologic — see the list below. (Biologics have no small-molecule generics.)
Why the date isn’t exact: Biosimilar timing can change because patents may be challenged, settled, licensed, added or removed, and litigation can move the real date earlier or later.
🛈 What do these terms mean?
- Biologic patent
- A patent the reference product’s maker has publicly listed. A biosimilar generally can’t launch until these expire — unless they’re invalidated or resolved in a settlement.
- Reference-product exclusivity
- A flat 12 years of FDA market protection from the biologic’s first licensure (the BPCIA). No biosimilar can be licensed before it ends, regardless of patents.
- Interchangeable exclusivity
- The first interchangeable biosimilar can earn a period as the only interchangeable version (pharmacists can substitute it without the prescriber).
- Earliest biosimilar (LOE)
- The latest of all the dates above — the soonest a biosimilar can realistically reach the market. Litigation and settlements can move it earlier.
Biologics have no small-molecule “generics” — competition comes from FDA-licensed biosimilars, tracked in the FDA Purple Book.
| Code | What it grants | Expires |
|---|---|---|
| RefProduct | Reference-product exclusivity (12-year, BPCIA) — no biosimilar can be licensed before this date | Jul 19, 2036 |
Is there a biosimilar for EPYSQLI 300 MG/30 ML VIAL?
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🔬 Reported adverse events (FAERS)
Top reported reactions
Age at onset
Reporter sex
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📦 Packaging — all sizes for this product
| Package NDC | Description | Marketing start | Status |
|---|---|---|---|
| 51759-0208-13 You're viewing this | 1 VIAL, SINGLE-DOSE in 1 CARTON (51759-208-13) / 30 mL in 1 VIAL, SINGLE-DOSE | 2025-04-07 | Active |
🧭 About this NDC listing & data coverage
What data is (and isn’t) available for this NDC — tap to expand
| NDC identity (package / product / labeler codes) | ✓ Available |
| Labeler | ✓ Available |
| Product & package description | ✓ Available |
| Marketing category & status | ✓ Available |
| Active ingredient / dosage form / route | ✓ Available |
| FDA label (SPL via DailyMed) | ✓ Available |
| Package photos | ✓ Available |
| Inactive ingredients (structured) | ✓ Available |
| NADAC pharmacy acquisition price (CMS) | — Not published for this NDC CMS publishes NADAC only for NDCs reported in its retail-pharmacy survey. |
| Orange Book / therapeutic-equivalence data | ✓ Available |
| HCPCS J-code billing crosswalk | ✓ Available |
| Medicaid utilization (CMS SDUD) | — Not published for this NDC CMS reports utilization only for NDCs with Medicaid claims above its privacy threshold. |
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📄 Full prescribing information FDA SPL
🚨 Boxed Warning ▾
WARNING: SERIOUS MENINGOCOCCAL INFECTIONS Eculizumab products, complement inhibitors, increase the risk of serious infections caused by Neisseria meningitidis [ see Warnings and Precautions ( 5.1 ) ]. Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early.
Complete or update vaccination for meningococcal bacteria (for serogroups A, C, W, Y, and B) at least 2 weeks prior to the first dose of EPYSQLI, unless the risks of delaying therapy with EPYSQLI outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against meningococcal bacteria in patients receiving a complement inhibitor. See Warnings and Precautions ( 5.1 ) for additional guidance on the management of the risk of serious infections caused by meningococcal bacteria.
Patients receiving eculizumab products are at increased risk for invasive disease caused by Neisseria meningitidis , even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious meningococcal infections and evaluate immediately if infection is suspected. Because of the risk of serious meningococcal infections, EPYSQLI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called EPYSQLI REMS [ see Warnings and Precautions ( 5.2 ) ].
WARNING: SERIOUS MENINGOCOCCAL INFECTIONS See full prescribing information for complete boxed warning. Eculizumab products increase the risk of serious and life-threatening infections caused by Neisseria meningitidis. Complete or update meningococcal vaccination at least 2 weeks prior to the first dose of EPYSQLI, unless the risks of delaying EPYSQLI outweigh the risk of developing a serious infection.
Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients receiving a complement inhibitor. ( 5.1 ) Patients receiving eculizumab products are at increased risk for invasive disease caused by N. meningitidis , even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of meningococcal infections, and evaluate immediately if infection is suspected.
( 5.1 ) EPYSQLI is available only through a restricted program called the EPYSQLI REMS. ( 5.2 )
🎯 Indications and Usage ▾
1 INDICATIONS AND USAGE EPYSQLI is a complement inhibitor indicated for: the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. ( 1.1 ) the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy. ( 1.2 ) Limitation of Use EPYSQLI is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.
( 1.3 ) the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. ( 1.4 )
1.1Paroxysmal Nocturnal Hemoglobinuria (PNH) EPYSQLI is indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis.
1.2Atypical Hemolytic Uremic Syndrome (aHUS) EPYSQLI is indicated for the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy. Limitation of Use EPYSQLI is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
1.3Generalized Myasthenia Gravis (gMG) EPYSQLI is indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.
1.4Neuromyelitis Optica Spectrum Disorder (NMOSD) EPYSQLI is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.
⏱️ Dosage and Administration ▾
2 DOSAGE AND ADMINISTRATION For intravenous infusion only; recommended dosage for: PNH: ( 2.2 ) aHUS, gMG, and NMOSD in adults: ( 2.3 ) aHUS in pediatric patients: ( 2.4 )
2.1Recommended Vaccination and Prophylaxis for Meningococcal Infection Vaccinate patients against meningococcal infection (serogroups A, C, W, Y, and B) according to current ACIP recommendations at least 2 weeks prior to initiation of EPYSQLI [ see Warnings and Precautions ( 5.1 ) ]. If urgent EPYSQLI therapy is indicated in a patient who is not up to date with meningococcal vaccines according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer these vaccines as soon as possible.
Healthcare providers who prescribe EPYSQLI must enroll in the EPYSQLI REMS [see Warnings and Precautions ( 5.2 )].
2.2Recommended Dosage for Adults – PNH The recommended dosage of EPYSQLI for the treatment of PNH in patients 18 years of age and older is administered as an intravenous infusion [see Dosage and Administration (2.7) ] as follows: 600 mg weekly for the first 4 weeks, followed by 900 mg for the fifth dose 1 week later, then 900 mg every 2 weeks thereafter. Administer EPYSQLI at the recommended dosage regimen time points, or within two days of these time points [ see Warnings and Precautions ( 5.4 ) ].
2.3Recommended Dosage for Adults – aHUS, gMG, and NMOSD The recommended dosage of EPYSQLI for the treatment of aHUS, gMG, or NMOSD in patients 18 years of age and older is administered as an intravenous infusion [see Dosage and Administration (2.7) ] as follows: 900 mg weekly for the first 4 weeks, followed by 1,200 mg for the fifth dose 1 week later, then 1,200 mg every 2 weeks thereafter.
2.4Recommended Dosage for Pediatric Patients – aHUS The recommended dosage of EPYSQLI for the treatment of aHUS in pediatric patients less than 18 years of age is administered as an intravenous infusion based upon body weight, according to the following schedule (Table 1): Table 1: Dosing Recommendations in Pediatric Patients Less Than 18 Years of Age with aHUS Patient Body Weight Induction Maintenance 40 kg and over 900 mg weekly for the first 4 weeks 1,200 mg at week 5; then 1,200 mg every 2 weeks 30 kg to less than 40 kg 600 mg weekly for the first 2 weeks 900 mg at week 3; then 900 mg every 2 weeks 20 kg to less than 30 kg 600 mg weekly for the first 2 weeks 600 mg at week 3; then 600 mg every 2 weeks 10 kg to less than 20 kg 600 mg single dose at Week 1 300 mg at week 2; then 300 mg every 2 weeks 5 kg to less than 10 kg 300 mg single dose at Week 1 300 mg at week 2; then 300 mg every 3 weeks Administer EPYSQLI at the recommended dosage regimen time points, or within two days of these time points.
2.5Dose Adjustment in Case of Plasmapheresis, Plasma Exchange, Fresh Frozen Plasma Infusion or IVIg For adult and pediatric patients with aHUS, and adult patients with gMG or NMOSD, supplemental dosing of EPYSQLI is required in the setting of concomitant plasmapheresis or plasma exchange, or fresh frozen plasma infusion (PE/PI) ( Table 2 ). Table 2: Supplemental Dose of EPYSQLI after Plasmapheresis/PE/PI Type of Plasma Intervention Most Recent EPYSQLI Dose Supplemental EPYSQLI Dose with Each Plasma Intervention Timing of Supplemental EPYSQLI Dose Plasmapheresis or plasma exchange 300 mg 300 mg per each plasmapheresis or plasma exchange session Within 60 minutes after each plasmapheresis or plasma exchange ≥600 mg 600 mg per each plasmapheresis or plasma exchange session Fresh frozen plasma infusion ≥300 mg 300 mg per infusion of fresh frozen plasma 60 minutes prior to each infusion of fresh frozen plasma For patients with gMG, a supplemental dose of EPYSQLI is required in the setting of concomitant use of intravenous immunoglobulin (IVIg) treatment as described in Table 3.
Table 3: Supplemental Dose of EPYSQLI with concomitant IVIg IVIg Frequency Most Recent EPYSQLI Dose Supplemental EPYSQLI Dose per IVIg Cycle Timing of Supplemental EPYSQLI D…
💊 Dosage Forms and Strengths ▾
3 DOSAGE FORMS AND STRENGTHS Injection: 300 mg/30 mL (10 mg/mL) as a clear to slightly opalescent and colorless solution in a single-dose vial. Injection: 300 mg/30 mL (10 mg/mL) in a single-dose vial. ( 3 )
⛔ Contraindications ▾
4 CONTRAINDICATIONS EPYSQLI is contraindicated for initiation in patients with unresolved serious Neisseria meningitidis infection [ see Warnings and Precautions ( 5.1 ) ]. EPYSQLI is contraindicated for initiation in patients with unresolved serious Neisseria meningitidis infection. ( 4 )
⚠️ Warnings and Cautions ▾
5 WARNINGS AND PRECAUTIONS Use caution when administering EPYSQLI to patients with any other systemic infection. ( 5.3 ) Infusion-Related Reactions: Monitor patients during infusion, interrupt for reactions, and institute appropriate supportive measures. ( 5.6 )
5.1Serious Meningococcal Infections Eculizumab products, complement inhibitors, increase a patient's susceptibility to serious, life-threatening, or fatal infections caused by meningococcal bacteria (septicemia and/or meningitis) in any serogroup, including non-groupable strains. Life-threatening and fatal meningococcal infections have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of EPYSQLI treatment is contraindicated in patients with unresolved serious Neisseria meningitidis infection.
Complete or update meningococcal vaccination (for serogroups A, C, W, Y, and B) at least 2 weeks prior to administration of the first dose of EPYSQLI, according to current ACIP recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations, considering the duration of therapy with EPYSQLI. Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information.
If urgent EPYSQLI therapy is indicated in a patient who is not up to date with meningococcal vaccines according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer meningococcal vaccines as soon as possible. Various durations and regimens of antibacterial drug prophylaxis have been considered, but the optimal durations and drug regimens for prophylaxis and their efficacy have not been studied in unvaccinated or vaccinated patients receiving complement inhibitors, including eculizumab products.
The benefits and risks of treatment with EPYSQLI, as well as the benefits and risks of antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by Neisseria meningitidis . Vaccination does not eliminate the risk of serious meningococcal infections, despite development of antibodies following vaccination. Closely monitor patients for early signs and symptoms of meningococcal infection and evaluate patients immediately if infection is suspected.
Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if these signs and symptoms occur. Promptly treat known infections. Meningococcal infection may become rapidly life- threatening or fatal if not recognized and treated early.
Consider interruption of EPYSQLI in patients who are undergoing treatment for serious meningococcal infection, depending on the risks of interrupting treatment in the disease being treated. EPYSQLI is available only through a restricted program under a REMS [see Warnings and Precautions ( 5.2 )] .
5.2EPYSQLI REMS EPYSQLI is available only through a restricted program under a REMS called EPYSQLI REMS, because of the risk of serious meningococcal infections [see Warnings and Precautions ( 5.1 )] . Notable requirements of the EPYSQLI REMS include the following: Prescribers must enroll in the REMS. Prescribers must counsel patients about the risk of serious meningococcal infection.
Prescribers must provide the patients with the REMS educational materials. Prescribers must assess patient vaccination status for meningococcal vaccines (against serogroups A, C, W, Y and B) and vaccinate if needed according to current ACIP recommendations two weeks prior to the first dose of EPYSQLI. Prescribers must provide a prescription for antibacterial drug prophylaxis if treatment must be started urgently and the patient is not up to date with meningococcal vaccines according to current ACIP recommendations at least two weeks prior to the first dose of EPYSQLI.
Healthcare settings and pharmacies…
🤒 Adverse Reactions ▾
6 ADVERSE REACTIONS The following serious adverse reactions are discussed in greater detail in other sections of the labeling: Serious Meningococcal Infections [ see Warnings and Precautions ( 5.1 ) ] Other Infections [ see Warnings and Precautions ( 5.3 ) ] Monitoring Disease Manifestations after EPYSQLI Discontinuation [ see Warnings and Precautions ( 5.4 ) ] Thrombosis Prevention and Management [ see Warnings and Precautions ( 5.5 ) ] Infusion-Related Reactions [ see Warnings and Precautions ( 5.6 ) ] The most frequently reported adverse reactions in the PNH randomized trial (≥10% overall and greater than placebo) are: headache, nasopharyngitis, back pain, and nausea.
( 6.1 ) The most frequently reported adverse reactions in aHUS single arm prospective trials (≥20%) are: headache, diarrhea, hypertension, upper respiratory infection, abdominal pain, vomiting, nasopharyngitis, anemia, cough, peripheral edema, nausea, urinary tract infections, pyrexia. ( 6.1 ) The most frequently reported adverse reaction in the gMG placebo-controlled clinical trial (≥10%) in adult patients is musculoskeletal pain. ( 6.1 ) The most frequently reported adverse reactions in the NMOSD placebo-controlled trial (≥10%) are: upper respiratory infection, nasopharyngitis, diarrhea, back pain, dizziness, influenza, arthralgia, pharyngitis, and contusion.
( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Teva Pharmaceuticals at 1-888-483-8279 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch .
6.1Clinical Trial Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Meningococcal infections are the most important adverse reactions experienced by patients receiving eculizumab. In PNH clinical studies, two patients experienced meningococcal sepsis.
Both patients had previously received a meningococcal vaccine. In clinical studies among patients without PNH, meningococcal meningitis occurred in one unvaccinated patient. Meningococcal sepsis occurred in one previously vaccinated patient enrolled in the retrospective aHUS study during the post-study follow-up period [ see Warnings and Precautions ( 5.1 ) ].
PNH The data described below reflect exposure to eculizumab in 196 adult patients with PNH, age 18-85, of whom 55% were female. All had signs or symptoms of intravascular hemolysis. Eculizumab was studied in a placebo-controlled clinical study (PNH Study 1, in which 43 patients received eculizumab and 44, placebo); a single arm clinical study (PNH Study 2); and a long term extension study (E05-001).
182 patients were exposed for greater than one year. All patients received the recommended eculizumab dose regimen. Table 6 summarizes the adverse reactions that occurred at a numerically higher rate in the eculizumab group than the placebo group and at a rate of 5% or more among patients treated with eculizumab.
Table 6: Adverse Reactions Reported in 5% or More of Eculizumab Treated Patients with PNH and Greater than Placebo in the Controlled Clinical Study Reaction Eculizumab Placebo (N=43) (N=44) N (%) N (%) Headache 19 (44) 12 (27) Nasopharyngitis 10 (23) 8 (18) Back pain 8 (19) 4 (9) Nausea 7 (16) 5 (11) Fatigue 5 (12) 1 (2) Cough 5 (12) 4 (9) Herpes simplex infections 3 (7) 0 Sinusitis 3 (7) 0 Respiratory tract infection 3 (7) 1 (2) Constipation 3 (7) 2 (5) Myalgia 3 (7) 1 (2) Pain in extremity 3 (7) 1 (2) Influenza-like illness 2 (5) 1 (2) In the placebo-controlled clinical study, serious adverse reactions occurred among 4 (9%) patients receiving eculizumab and 9 (21%) patients receiving placebo.
The serious reactions included infections and progression of PNH. No deaths occurred in the study and no patients receiving eculizumab experienced a thrombotic event; one thrombotic event occurred in a patient receiving placebo. Among 193 pat…
🔄 Drug Interactions ▾
7 DRUG INTERACTIONS
7.1Plasmapheresis, Plasma Exchange, Fresh Frozen Plasma Infusion or IVIg Concomitant use of eculizumab products with plasma exchange (PE), plasmapheresis (PP), fresh frozen plasma infusion (PE/PI), or in patients with gMG on concomitant IVIg treatment can reduce serum eculizumab product concentrations and requires a supplemental dose of EPYSQLI [see Dosage and Administration ( 2.5 )] .
7.2Neonatal Fc Receptor Blockers Concomitant use of eculizumab products with neonatal Fc receptor (FcRn) blockers may lower systemic exposures and reduce effectiveness of eculizumab products. Closely monitor for reduced effectiveness of EPYSQLI.
👥 Use in Specific Populations ▾
8 USE IN SPECIFIC POPULATIONS
8.1Pregnancy Risk Summary Limited data on outcomes of pregnancies that have occurred following eculizumab use in pregnant women have not identified a concern for specific adverse developmental outcomes (see Data). There are risks to the mother and fetus associated with untreated paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in pregnancy ( see Clinical Considerations ). Animal studies using a mouse analogue of the eculizumab molecule (murine anti-C5 antibody) showed increased rates of developmental abnormalities and an increased rate of dead and moribund offspring at doses 2-8 times the human dose ( see Data ).
The estimated background risk of major birth defects and miscarriage for the indicated populations is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defect and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively.
Clinical Considerations Disease-Associated Maternal and/or Fetal/Neonatal Risk PNH in pregnancy is associated with adverse maternal outcomes, including worsening cytopenias, thrombotic events, infections, bleeding, miscarriages and increased maternal mortality, and adverse fetal outcomes, including fetal death and premature delivery. aHUS in pregnancy is associated with adverse maternal outcomes, including pre-eclampsia and preterm delivery, and adverse fetal/neonatal outcomes, including intrauterine growth restriction (IUGR), fetal death and low birth weight.
Data Human Data A pooled analysis of prospectively (50.3%) and retrospectively (49.7%) collected data in more than 300 pregnant women with live births following exposure to eculizumab have not suggested safety concerns. However, these data cannot definitively exclude any drug-associated risk during pregnancy, because of the limited sample size. Animal Data Animal reproduction studies were conducted in mice using doses of a murine anti-C5 antibody that approximated 2-4 times (low dose) and 4-8 times (high dose) the recommended human eculizumab dose, based on a body weight comparison.
When animal exposure to the antibody occurred in the time period from before mating until early gestation, no decrease in fertility or reproductive performance was observed. When maternal exposure to the antibody occurred during organogenesis, two cases of retinal dysplasia and one case of umbilical hernia were observed among 230 offspring born to mothers exposed to the higher antibody dose; however, the exposure did not increase fetal loss or neonatal death. When maternal exposure to the antibody occurred in the time period from implantation through weaning, a higher number of male offspring became moribund or died (1/25 controls, 2/25 low dose group, 5/25 high dose group).
Surviving offspring had normal development and reproductive function.
8.2Lactation Risk Summary Although limited published data does not report detectable levels of eculizumab in human milk, maternal IgG is known to be present in human milk. Available information is insufficient to inform the effect of eculizumab products on the breastfed infant. There are no data on the effects of eculizumab products on milk production.
The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for EPYSQLI and any potential adverse effects on the breastfed child from EPYSQLI or from the underlying maternal condition.
8.4Pediatric Use PNH and NMOSD Safety and effectiveness of EPYSQLI for the treatment of PNH, or NMOSD in pediatric patients have not been established. aHUS The safety and effectiveness of EPYSQLI for the treatment of aHUS have been established in pediatric patients. Use of EPYSQLI in pediatric patients for this indication is supported by evidence from four adequate and well-controlled clinical studies assessing the safety and e…
🤰 Pregnancy ▾
8.1Pregnancy Risk Summary Limited data on outcomes of pregnancies that have occurred following eculizumab use in pregnant women have not identified a concern for specific adverse developmental outcomes (see Data). There are risks to the mother and fetus associated with untreated paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in pregnancy ( see Clinical Considerations ). Animal studies using a mouse analogue of the eculizumab molecule (murine anti-C5 antibody) showed increased rates of developmental abnormalities and an increased rate of dead and moribund offspring at doses 2-8 times the human dose ( see Data ).
The estimated background risk of major birth defects and miscarriage for the indicated populations is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defect and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively.
Clinical Considerations Disease-Associated Maternal and/or Fetal/Neonatal Risk PNH in pregnancy is associated with adverse maternal outcomes, including worsening cytopenias, thrombotic events, infections, bleeding, miscarriages and increased maternal mortality, and adverse fetal outcomes, including fetal death and premature delivery. aHUS in pregnancy is associated with adverse maternal outcomes, including pre-eclampsia and preterm delivery, and adverse fetal/neonatal outcomes, including intrauterine growth restriction (IUGR), fetal death and low birth weight.
Data Human Data A pooled analysis of prospectively (50.3%) and retrospectively (49.7%) collected data in more than 300 pregnant women with live births following exposure to eculizumab have not suggested safety concerns. However, these data cannot definitively exclude any drug-associated risk during pregnancy, because of the limited sample size. Animal Data Animal reproduction studies were conducted in mice using doses of a murine anti-C5 antibody that approximated 2-4 times (low dose) and 4-8 times (high dose) the recommended human eculizumab dose, based on a body weight comparison.
When animal exposure to the antibody occurred in the time period from before mating until early gestation, no decrease in fertility or reproductive performance was observed. When maternal exposure to the antibody occurred during organogenesis, two cases of retinal dysplasia and one case of umbilical hernia were observed among 230 offspring born to mothers exposed to the higher antibody dose; however, the exposure did not increase fetal loss or neonatal death. When maternal exposure to the antibody occurred in the time period from implantation through weaning, a higher number of male offspring became moribund or died (1/25 controls, 2/25 low dose group, 5/25 high dose group).
Surviving offspring had normal development and reproductive function.
🧒 Pediatric Use ▾
8.4Pediatric Use PNH and NMOSD Safety and effectiveness of EPYSQLI for the treatment of PNH, or NMOSD in pediatric patients have not been established. aHUS The safety and effectiveness of EPYSQLI for the treatment of aHUS have been established in pediatric patients. Use of EPYSQLI in pediatric patients for this indication is supported by evidence from four adequate and well-controlled clinical studies assessing the safety and effectiveness of eculizumab for the treatment of aHUS. The studies included a total of 47 pediatric patients (ages 2 months to 17 years).
The safety and effectiveness of eculizumab for the treatment of aHUS appear similar in pediatric and adult patients [ see Adverse Reactions ( 6.1 ), and Clinical Studies ( 14.2 ) ]. A pediatric assessment for EPYSQLI demonstrates that EPYSQLI is safe and effective for pediatric patients in an indication for which Soliris (eculizumab) is approved. However, EPYSQLI is not approved for such indication due to marketing exclusivity for Soliris (eculizumab).
Vaccinations Administer vaccinations for the prevention of infection due to Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) according to ACIP guidelines [ see Warnings and Precautions ( 5.1 , 5.3 ) ].
🧓 Geriatric Use ▾
8.5Geriatric Use Fifty-one patients 65 years of age or older (15 with PNH, 4 with aHUS, 26 with gMG, and 6 with NMOSD) were treated with eculizumab in clinical trials in the approved indications. Although there were no apparent age-related differences observed in these studies, the number of patients aged 65 and over is not sufficient to determine whether they respond differently from younger patients.
🧬 Clinical Pharmacology ▾
12 CLINICAL PHARMACOLOGY
12.1Mechanism of Action Eculizumab-aagh, the active ingredient in EPYSQLI, is a monoclonal antibody that specifically binds to the complement protein C5 with high affinity, thereby inhibiting its cleavage to C5a and C5b and preventing the generation of the terminal complement complex C5b-9. Eculizumab products inhibit terminal complement-mediated intravascular hemolysis in PNH patients and complement-mediated thrombotic microangiopathy (TMA) in patients with aHUS. The precise mechanism by which eculizumab products exert their therapeutic effect in gMG patients is unknown, but is presumed to involve reduction of terminal complement complex C5b-9 deposition at the neuromuscular junction.
The precise mechanism by which eculizumab products exert their therapeutic effect in NMOSD is unknown, but is presumed to involve inhibition of aquaporin-4-antibody induced terminal complement C5b-9 deposition.
12.2Pharmacodynamics In the placebo-controlled clinical study (PNH Study 1), eculizumab when administered as recommended reduced serum LDH levels from 2200 ± 1034 U/L (mean ± SD) at baseline to 700 ± 388 U/L by week one and maintained the effect through the end of the study at week 26 (327 ± 433 U/L) in patients with PNH. In the single arm clinical study (PNH Study 2), the effect was maintained through week 52 [ see Clinical Studies ( 14 ) ]. In patients with PNH, aHUS, gMG, and NMOSD, free C5 concentrations of < 0.5 mcg/mL was correlated with complete blockade of terminal complement activity.
12.3Pharmacokinetics Following intravenous maintenance doses of 900 mg once every 2 weeks in patients with PNH, the week 26 observed mean ± SD serum eculizumab maximum concentration (C max ) was 194 ± 76 mcg/mL and the trough concentration (C trough ) was 97 ± 60 mcg/mL. Following intravenous maintenance doses of 1,200 mg once every 2 weeks in patients with aHUS, the week 26 observed mean ± SD C trough was 242 ± 101 mcg/mL. Following intravenous maintenance doses of 1,200 mg once every 2 weeks in adult patients with gMG, the week 26 observed mean ± SD C max was 783 ± 288 mcg/mL and the C trough was 341 ± 172 mcg/mL.
Following intravenous maintenance doses of 1200 mg once every 2 weeks in patients with NMOSD, at week 24, the observed mean ± SD C max was 877 ± 331 and the C trough was 429 ± 188 mcg/mL. Steady state was achieved 4 weeks after starting eculizumab treatment, with accumulation ratio of approximately 2-fold in all studied indications. Population pharmacokinetic analyses showed that eculizumab pharmacokinetics were dose-linear and time-independent over the 600 mg to 1,200 mg dose range, with inter-individual variability of 21% to 38%.
Distribution The eculizumab volume of distribution for a typical 70 kg patient was 5 L to 8 L. Elimination The half-life of eculizumab ranged between 141 h and 882 h. Plasma exchange or infusion increased the clearance of eculizumab by approximately 6.01-fold and reduced the half-life to 90.2 h.
Supplemental dosing is recommended when EPYSQLI is administered to patients receiving plasma exchange or infusion [ see Dosage and Administration ( 2.5 ) ]. Specific Populations Age, Sex, and Race The pharmacokinetics of eculizumab were not affected by age (2 months to 85 years), sex, or race. Renal Impairment Renal function did not affect the pharmacokinetics of eculizumab in PNH (creatinine clearance of 8 mL/min to 396 mL/min calculated using Cockcroft-Gault formula), aHUS (estimated glomerular filtration rate [eGFR] of 5 mL/min/1.73 m 2 to105 mL/min/1.73 m 2 using the Modification of Diet in Renal Disease [MDRD] formula), or gMG patients (eGFR of 44 mL/min/1.73 m 2 to 168 mL/min/1.73 m 2 using MDRD formula).
12.6Immunogenicity The observed incidence of anti-drug antibodies is highly dependent on the sensitivity and specificity of the assay. Differences in assay methods preclude meaningful comparisons of the incidence of anti-drug antibodies in the studies described below with the inciden…
🧬 Mechanism of Action ▾
12.1Mechanism of Action Eculizumab-aagh, the active ingredient in EPYSQLI, is a monoclonal antibody that specifically binds to the complement protein C5 with high affinity, thereby inhibiting its cleavage to C5a and C5b and preventing the generation of the terminal complement complex C5b-9. Eculizumab products inhibit terminal complement-mediated intravascular hemolysis in PNH patients and complement-mediated thrombotic microangiopathy (TMA) in patients with aHUS. The precise mechanism by which eculizumab products exert their therapeutic effect in gMG patients is unknown, but is presumed to involve reduction of terminal complement complex C5b-9 deposition at the neuromuscular junction.
The precise mechanism by which eculizumab products exert their therapeutic effect in NMOSD is unknown, but is presumed to involve inhibition of aquaporin-4-antibody induced terminal complement C5b-9 deposition.
📦 How Supplied / Storage and Handling ▾
16 HOW SUPPLIED/STORAGE AND HANDLING EPYSQLI (eculizumab-aagh) injection is a sterile, preservative-free, clear to slightly opalescent and colorless solution supplied as one 300 mg/30 mL (10 mg/mL) single-dose vial per carton (NDC 51759-208-13). Store EPYSQLI vials in a refrigerator at 2°C to 8°C (36°F to 46°F) in the original carton to protect from light until time of use. DO NOT FREEZE.
DO NOT SHAKE. Prior to administration, if needed, unopened vials of EPYSQLI may be stored in the original carton at controlled room temperature [not more than 30°C (86°F)] for a single 1 month period and then returned to refrigeration for 3 days. Do not use beyond the expiration date shown on the carton.
Refer to Dosage and Administration ( 2 ) for information on the stability and storage of diluted solutions of EPYSQLI.
📋 Description ▾
11 DESCRIPTION Eculizumab-aagh, a complement inhibitor, is a recombinant humanized monoclonal IgG2/4 κ antibody produced in a Chinese Hamster Ovary cell line expression system and purified by standard bioprocess technology . Eculizumab-aagh contains human constant regions from human IgG2 sequences and human IgG4 sequences and murine complementarity-determining regions grafted onto the human framework light- and heavy-chain variable regions. Eculizumab-aagh is composed of two 448 amino acid heavy chains and two 214 amino acid light chains and has a molecular weight of approximately 148 kDa.
EPYSQLI (eculizumab-aagh) injection is a sterile, preservative-free, clear to slightly opalescent and colorless solution for intravenous infusion and is supplied in a 30 mL single-dose vial. Each mL contains 10 mg of eculizumab-aagh, dibasic sodium phosphate (0.77 mg), monobasic sodium phosphate (0.55 mg), polysorbate 80 (0.22 mg) (vegetable origin), trehalose (86 mg), and Water for Injection, USP. The pH is 7.
💬 Information for Patients ▾
17 PATIENT COUNSELING INFORMATION Advise the patients and/or caregivers to read the FDA-approved patient labeling ( Medication Guide ). Serious Meningococcal Infections Advise patients of the risk of serious meningococcal infection. Inform patients of the need to complete or update their meningococcal vaccinations at least 2 weeks prior to receiving the first dose of EPYSQLI or receive antibacterial drug prophylaxis if EPYSQLI treatment must be initiated immediately and they have not been previously vaccinated.
Inform patients of the requirement to be revaccinated according to current ACIP recommendations for meningococcal infection while on EPYSQLI therapy [see Warnings and Precautions ( 5.1 )] . Inform patients that vaccination may not prevent serious meningococcal infection and to seek immediate medical attention if the following signs or symptoms occur [see Warnings and Precautions ( 5.1 )] : fever fever and a rash fever with high heart rate headache with nausea or vomiting headache and a fever headache with a stiff neck or stiff back confusion muscle aches with flu-like symptoms eyes sensitive to light Inform patients that they will be given a Patient Safety Card for EPYSQLI that they should carry with them at all times during and for 3 months following treatment with EPYSQLI.
This card describes symptoms which, if experienced, should prompt the patient to immediately seek medical evaluation. EPYSQLI REMS EPYSQLI is available only through a restricted program called EPYSQLI REMS [see Warnings and Precautions ( 5.2 )] . Inform the patient of the following notable requirements: Patients must receive counseling about the risk of serious meningococcal infections.
Patients must receive written educational materials about this risk. Patients must be instructed to carry the Patient Safety Card with them at all times during and for 3 months following treatment with EPYSQLI. Patients must be instructed to complete or update meningococcal vaccines for serogroups A, C, W, Y and B per ACIP recommendations as directed by the prescriber prior to treatment with EPYSQLI.
Patients must receive antibiotics as directed by the prescriber if they are not up to date with meningococcal vaccines and have to start EPYSQLI right away. Other Infections Counsel patients about gonorrhea prevention and advise regular testing for patients at-risk. Inform patients that there may be an increased risk of other types of infections, particularly those due to encapsulated bacteria.
Aspergillus infections have occurred in immunocompromised and neutropenic patients. Inform parents or caregivers of children receiving EPYSQLI for the treatment of aHus that their child should be vaccinated against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) according to current medical guidelines. Infusion-Related Reactions Advise patients that administration of EPYSQLI may result in infusion-related reactions.
Discontinuation Inform patients with PNH that they may develop serious hemolysis due to PNH when EPYSQLI is discontinued and that they will be monitored by their healthcare professional for at least 8 weeks following EPYSQLI discontinuation. Inform patients with aHUS that there is a potential for TMA complications due to aHUS when EPYSQLI is discontinued and that they will be monitored by their healthcare professional for at least 12 weeks following EPYSQLI discontinuation. Inform patients who discontinue EPYSQLI to keep the Patient Safety Card with them for three months after the last EPYSQLI dose, because the increased risk of meningococcal infection persists for several weeks following discontinuation of EPYSQLI.
Manufactured by: Samsung Bioepis Co., Ltd. 76, Songdogyoyuk-ro, Yeonsu-gu, Incheon, 21987 Republic of Korea US License Number 2046 Manufactured for: Teva Pharmaceuticals Parsippany, NJ 07054
💬 Medication Guide ▾
This Medication Guide has been approved by the U.S. Food and Drug Administration. Revised: 06/2026 MEDICATION GUIDE EPYSQLI ® (eh-pis'-klee) (eculizumab-aagh) injection, for intravenous use What is the most important information I should know about EPYSQLI?
EPYSQLI is a medicine that affects your immune system. EPYSQLI may lower the ability of your immune system to fight infections. EPYSQLI increases your chance of getting serious meningococcal infections caused by Neisseria meningitidis bacteria.
Meningococcal infections may quickly become life-threatening or cause death if not recognized and treated early. You must complete or update your meningococcal vaccine(s) at least 2 weeks before your first dose of EPYSQLI. If you have not completed your meningococcal vaccines and EPYSQLI must be started right away, you should receive the required vaccine(s) as soon as possible.
If you have not been vaccinated and EPYSQLI must be started right away, you should also receive antibiotics to take for as long as your healthcare provider tells you. If you had a meningococcal vaccine in the past, you might need additional vaccines before starting EPYSQLI. Your healthcare provider will decide if you need additional meningococcal vaccines.
Meningococcal vaccines do not prevent all meningococcal infections. Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a serious meningococcal infection : fever fever with high heart rate headache and fever confusion muscle aches with flu-like symptoms fever and a rash headache with nausea or vomiting headache with a stiff neck or stiff back eyes sensitive to light Your healthcare provider will give you a Patient Safety Card about the risk of serious meningococcal infection.
Carry it with you at all times during treatment and for 3 months after your last dose of EPYSQLI. Your risk of meningococcal infection may continue for several weeks after your last dose of EPYSQLI. It is important to show this card to any healthcare provider who treats you.
This will help them diagnose and treat you quickly. EPYSQLI is only available through a program called the EPYSQLI Risk Evaluation and Mitigation Strategy (REMS). Before you can receive EPYSQLI, your healthcare provider must: enroll in the EPYSQLI REMS program counsel you about the risk of serious meningococcal infections give you information about the signs and symptoms of serious meningococcal infection make sure that you are vaccinated against serious infections caused by meningococcal bacteria and that you receive antibiotics if you need to start EPYSQLI right away and you are not up to date on your vaccines give you a Patient Safety Card about your risk of meningococcal infection, as discussed above EPYSQLI may also increase the risk of other types of serious infections caused by encapsulated bacteria, including Streptococcus pneumoniae , Haemophilus influenzae , and Neisseria gonorrhoeae .
If your child is treated with EPYSQLI, your child should receive vaccines against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib). Certain people may be at risk of serious infections with gonorrhea. Talk to your healthcare provider about whether you are at risk for gonorrhea infection, about gonorrhea prevention, and regular testing.
Certain fungal infections (aspergillus) may also happen if you take EPYSQLI and have a weak immune system or a low white blood cell count. For more information about side effects, see " What are the possible side effects of EPYSQLI? " What is EPYSQLI? EPYSQLI is a prescription medicine used to treat: people with paroxysmal nocturnal hemoglobinuria (PNH). people with atypical hemolytic uremic syndrome (aHUS).
EPYSQLI is not for use in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. adults with neuromyelitis optica spect…