Zilucoplan

Zilucoplan binds to the complement protein C5 and inhibits its cleavage to C5a and C5b, preventing the generation of the terminal complement complex, C5b-9. The precise mechanism by which zilucoplan exerts its therapeutic effect in generalized myasthenia gravis is unknown but is presumed to involve reduction of C5b-9 deposition at the neuromuscular junction.

• ZILBRYSQ is indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. ZILBRYSQ is a complement inhibitor indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.

• ZILBRYSQ is contraindicated for initiation in patients with unresolved serious Neisseria meningitidis infection [see Warnings and Precautions (5.1) ] . ZILBRYSQ is contraindicated for initiation in patients with unresolved serious Neisseria meningitidis infection.contraindicated

Obtain baseline amylase and lipase. ( 2.2 ) For subcutaneous injection only. ( 2.3 ) Recommended dosage ( 2.3 ): Body Weight Once Daily Dosage Plunger Rod Color of Prefilled Syringe Less than 56 kg 16.6 mg RUBINE RED 56 kg to less than 77 kg 23 mg ORANGE 77 kg and above 32.4 mg DARK BLUE See Full Prescribing Information for instructions on dosage, preparation, and administration. ( 2.4 , 2.5 ) 2.1 Recommended 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 ZILBRYSQ [see Warnings and Precautions (5.1) ] . If urgent ZILBRYSQ 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 [see Warnings and Precautions (5.1) ] . Healthcare providers who prescribe ZILBRYSQ must enroll in the ZILBRYSQ REMS [see Warnings and Precautions (5.2) ] . 2.2 Recommended Testing Before Initiating ZILBRYSQ Before initiating ZILBRYSQ, obtain baseline lipase and amylase levels [see Warnings and Precautions (5.4) ] . 2.3 Recommended Dosage The recommended dosage of ZILBRYSQ is given once daily as a subcutaneous injection and is dependent on actual body weight (see Table 1 ).

Other Infections: Use caution when administering ZILBRYSQ to patients with any other systemic infection. ( 5.3 ) Pancreatitis and pancreatic cysts have been reported in patients treated with ZILBRYSQ. Discontinue ZILBRYSQ in patients with suspected pancreatitis and initiate appropriate management until pancreatitis is ruled out or has resolved. ( 5.4 ) 5.1 Serious Meningococcal Infections ZILBRYSQ, a complement inhibitor, increases 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 ZILBRYSQ 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 ZILBRYSQ, according to current ACIP recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations considering the duration of ZILBRYSQ therapy. Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information.

The following clinically significant 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) ] Pancreatitis and Other Pancreatic Conditions [see Warnings and Precautions (5.4) ] The most common adverse reactions (≥10%) in patients with gMG were injection site reactions, upper respiratory tract infection, and diarrhea. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact UCB, Inc. at 844-599-2273 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials 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. A total of 212 patients were treated with ZILBRYSQ 0.3 mg/kg in clinical studies in gMG. Of these, 137 patients were exposed for at least 6 months, and 87 were exposed for at least 1 year. In a placebo-controlled study (Study 1) in patients with gMG, 86 patients received ZILBRYSQ 0.3 mg/kg [see Clinical Studies (14) ] . Of these 86 patients, approximately 61% were female, 77% were White, 8% were Asian, and 8% were of Hispanic or Latino ethnicity. The mean age at study entry was 52.6 years (range 21 to 75 years). Table 2 summarizes the adverse reactions reported in at least 5% of patients treated with ZILBRYSQ and more frequently than placebo.

Pregnancy: Based on animal data, may cause fetal harm. ( 8.1 ) 8.1 Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to ZILBRYSQ during pregnancy. Patients or healthcare providers may contact UCBCares at 1-844-599-CARE (2273) or email ucbcares@ucb.com, so that information about the exposure of ZILBRYSQ during pregnancy and/or breastfeeding can be collected. Risk Summary There are no available data on ZILBRYSQ use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. Administration of zilucoplan to pregnant monkeys resulted in increases in embryofetal death at maternal exposures similar to those in humans at therapeutic doses (see Animal Data ) . All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. The background rate of major birth defects and miscarriage in the indicated population is unknown. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

Metabolism

The population pharmacokinetics (PK) analysis showed that zilucoplan PK was not time dependent. Following single daily subcutaneous administration of ZILBRYSQ (0.05 mg/kg, 0.1 mg/kg, 0.2 mg/kg, and 0.4 mg/kg) in healthy subjects, increase in peak plasma concentration was approximately dose proportional and increase in area under the curve was less than dose proportional.

• Oct 17, 2023NDANDA216834Ucb Inc

1. 1Myasthenia Gravis25938%
2. 2Drug Ineffective14622%
3. 3Product Dose Omission Issue9915%
4. 4Injection Site Pain7711%
5. 5Fatigue608.8%
6. 6Diarrhoea517.5%
7. 7Hospitalisation497.2%
8. 8Myasthenia Gravis Crisis345.0%
9. 9Dyspnoea314.6%
10. 10Weight Decreased314.6%
11. 11Therapy Interrupted304.4%
12. 12Weight Increased294.3%
13. 13Nausea284.1%
14. 14Pneumonia284.1%
15. 15Syringe Issue284.1%

The 10 most recently updated of 19 ClinicalTrials.gov registrations naming Zilucoplan as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• HEALEY ALS Platform Trial - Master Protocol
  Recruiting · Phase 2 · Phase 3 · Interventional · 1,500 enrolled · Merit E. Cudkowicz, MD
  NCT04297683updated 2026-06-04
• An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
  Enrolling by invitation · Phase 3 · Interventional · 8 enrolled · UCB Biopharma SRL
  NCT06435312updated 2026-05-08
• A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
  Recruiting · Phase 2 · Phase 3 · Interventional · 8 enrolled · UCB Biopharma SRL
  NCT06055959updated 2026-05-08
• Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis
  Active not recruiting · Phase 3 · Interventional · 200 enrolled · UCB Biopharma SRL
  NCT04225871updated 2026-04-24
• A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis
  Completed · Phase 3 · Interventional · 31 enrolled · UCB Biopharma SRL
  NCT06471361updated 2026-03-27
• A Study to Assess Zilucoplan Concentration in Breast Milk of Healthy Lactating Women
  Recruiting · Phase 1 · Interventional · 15 enrolled · UCB Biopharma SRL
  NCT06961747updated 2026-03-24
• Zilucoplan for Severe gMG Exacerbations
  Recruiting · Phase 3 · Interventional · 15 enrolled · Miriam Freimer
  NCT07215949updated 2026-02-23
• A Study to Evaluate Zilucoplan Injected Subcutaneously Either by a Prefilled Syringe or an Auto-injector in Healthy Adult Participants
  Completed · Phase 1 · Interventional · 14 enrolled · UCB Biopharma SRL
  NCT06511076updated 2026-01-06
• Effectiveness and Safety of Zilucoplan Treatment for French Patients With Myasthenia Gravis Over 3 Months
  Recruiting · Observational · 55 enrolled · Centre Hospitalier Universitaire de Nice
  NCT06815133updated 2025-12-03
• An Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Zilucoplan in Participants With Generalized Myasthenia Gravis Who Were Previously Receiving Intravenous Complement Component 5 Inhibitors
  Completed · Phase 3 · Interventional · 26 enrolled · UCB Biopharma SRL
  NCT05514873updated 2025-09-03

Frequently asked questions

How does Zilucoplan work?

Zilucoplan binds to the complement protein C5 and inhibits its cleavage to C5a and C5b, preventing the generation of the terminal complement complex, C5b-9. The precise mechanism by which zilucoplan exerts its therapeutic effect in generalized myasthenia gravis is unknown but is presumed to involve reduction of C5b-9 deposition at the neuromuscular junction.

What is Zilucoplan used for?

According to FDA labeling, Zilucoplan carries indications including: ZILBRYSQ is indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. ZILBRYSQ is a complement inhibitor indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Zilucoplan?

Zilucoplan is classified as Complement inhibitors, Complement Inhibitor, Complement Inhibitors, Decreased Complement Degradation.

What are the brand names for Zilucoplan?

Zilucoplan is marketed under brand names including Zilbrysq.

What are the contraindications for Zilucoplan?

Zilucoplan labeling lists contraindications including: ZILBRYSQ is contraindicated for initiation in patients with unresolved serious Neisseria meningitidis infection [see Warnings and Precautions (5.1) ] . ZILBRYSQ is contraindicated for initiation in patients with unresolved serious Neisseria meningitidis infection.. Always consult the full prescribing information and a clinician.