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WARNING
SERIOUS MENINGOCOCCAL INFECTIONS
Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors; ZILBRYSQ is a complement inhibitor. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early [see WARNINGS AND PRECAUTIONS].
Because of the risk of serious meningococcal infections, ZILBRYSQ is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called ZILBRYSQ REMS [see WARNINGS AND PRECAUTIONS].
Zilucoplan, a complement inhibitor, is a 15 amino-acid, synthetic macrocyclic peptide. The molecular formula of zilucoplan is C172H278N24O55 in free acid form and its molecular weight is 3562.23 Daltons (free acid form).
The chemical name for zilucoplan sodium is: acetyl-[L-lysyl1-L-valyl2-L-glutamyl3-Larginyl4- L phenylalanyl5-L aspartyl6]-N-methyl L-aspartyl7-L tert-leucyl8-L tyrosyl9-L-7- azatryptophyl10-L glutamyl11-L-tyrosyl12-L prolyl13-L cyclohexylglycyl14-[L-lysyl15, Nε-palmitoyl-γ-L-glutamyl-(1-amino-3,6,9,12,15,18,21,24,27,30,33,36,39,42,45,48,51,54,57,60,63,66,69,72- tetracosaoxapentaheptacontan-75-oyl)], cyclic (Lactam 1-6), tetra sodium.
The primary structure for zilucoplan sodium is shown below:
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ZILBRYSQ injection is a sterile, clear to slightly opalescent, colorless, preservative-free, buffered solution of zilucoplan (as zilucoplan sodium) for subcutaneous injection, in single-dose prefilled syringes. The solution pH is between 6.5 and 7.5. ZILBRYSQ is supplied in three dose strengths containing 16.6 mg /0.416 mL, 23 mg /0.574 mL, and 32.4 mg/0.81 mL of zilucoplan free acid equivalent to 17 mg, 23.6 mg, and 33.2 mg of zilucoplan sodium, respectively. Additionally, each mL of the solution contains dibasic sodium phosphate, anhydrous (4.11 mg); monobasic sodium phosphate, monohydrate (2.9 mg); sodium chloride (4.42 mg); and water for injection.
ZILBRYSQ is indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.
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].
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].
Healthcare providers who prescribe ZILBRYSQ must enroll in the ZILBRYSQ REMS [see WARNINGS AND PRECAUTIONS].
Before initiating ZILBRYSQ, obtain baseline lipase and amylase levels [see WARNINGS AND PRECAUTIONS].
The recommended dosage of ZILBRYSQ is given once daily as a subcutaneous injection and is dependent on actual body weight (see Table 1).
Table 1: Total Daily Dosage by Body Weight Range
| 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 |
Injection: 16.6 mg/0.416 mL, 23 mg/0.574 mL, or 32.4 mg/0.81 mL of zilucoplan as a clear to slightly opalescent, colorless solution in single-dose prefilled syringes.
ZILBRYSQ (zilucoplan) injection prefilled syringe contains a sterile, preservative-free, clear to slightly opalescent, colorless solution. Each single-dose prefilled syringe consists of a 1 mL glass syringe with a 29-gauge ½ inch needle, a needle safety guard, and a needle cover. The ZILBRYSQ prefilled syringe components are not made with natural rubber latex.
ZILBRYSQ is available as follows:
| NDC | Carton Pack Size | Strength | Plunger Color |
| 50474-990-80 | 28 single-dose prefilled syringes (4 cartons each containing 7 syringes for a total of 28 syringes) | 16.6 mg/0.416 mL | RUBINE RED |
| 50474-991-80 | 28 single-dose prefilled syringes (4 cartons each containing 7 syringes for a total of 28 syringes) | 23 mg/0.574 mL | ORANGE |
| 50474-992-80 | 28 single-dose prefilled syringes (4 cartons each containing 7 syringes for a total of 28 syringes) | 32.4 mg/0.81 mL | DARK BLUE |
Store ZILBRYSQ prefilled syringes refrigerated at 2°C to 8°C (36°F to 46°F) in the original carton until dispensing. Do not freeze.
Storage conditions after dispensing by pharmacist are summarized in Table 4:
Table 4: After Dispensing Storage Conditions
| Temperature | Refrigeration 2°C to 8°C (36°F to 46°F) | Room Temperature Up to 30°C (86°F) |
| Time Period | Until expiration date on the carton | Up to 3 months after removing from refrigerator or until expiration date on the carton, whichever occurs first |
Manufactured for: UCB, Inc. 1950 Lake Park Drive Smyrna, GA 30080 Revised: Feb 2025
The following clinically significant adverse reactions are discussed in greater detail in other sections of the labeling:
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]. 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. The most common adverse reactions (reported in at least 10% of patients treated with ZILBRYSQ) were injection site reactions, upper respiratory tract infections, and diarrhea.
Table 2: Adverse Reactions in at least 5% of Patients Treated with ZILBRYSQ and More Frequently than in Patients who Received Placebo in Study 1
| Adverse Reaction | ZILBRYSQ 0.3 mg/kg (n=86)% |
Placebo (n=88)% |
| Injection site reactions | 29 | 16 |
| Upper respiratory tract infections | 14 | 7 |
| Diarrhea | 11 | 2 |
| Urinary tract infection | 8 | 5 |
| Nausea or vomiting | 8 | 7 |
| Lipase increased | 7 | 0 |
| Amylase increased | 5 | 1 |
In addition to increases in amylase and lipase observed in Study 1, pancreatic events, including pancreatitis and pancreatic cysts have been observed in patients taking ZILBRYSQ [see WARNINGS AND PRECAUTIONS].
Additional laboratory abnormalities included transient elevations of blood eosinophils, which were of uncertain clinical significance.
Morphea
In the open-label extension studies, which included 213 patients, morphea was observed in 10 (5%) patients; most cases had a time to onset longer than one year after start of treatment and were mild to moderate in severity. One patient discontinued ZILBRYSQ because of morphea.
No Information provided
Included as part of the PRECAUTIONS section.
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.
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 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 ZILBRYSQ. The benefits and risks of treatment with ZILBRYSQ, 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 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 ZILBRYSQ in patients who are undergoing treatment for serious meningococcal infection, depending on the risks of interrupting treatment in the disease being treated [see CONTRAINDICATIONS].
ZILBRYSQ is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) [see WARNINGS AND PRECAUTIONS].
ZILBRYSQ is available only through a restricted program under a REMS called ZILBRYSQ REMS, because of the risk of serious meningococcal infections [see WARNINGS AND PRECAUTIONS]. Notable requirements of the ZILBRYSQ REMS include the following:
Further information is available at www.ZILBRYSQREMS.com or 1-877-414-8353.
Serious infections with Neisseria species (other than Neisseria meningitidis), including disseminated gonococcal infections, have been reported in patients treated with complement inhibitors.
ZILBRYSQ blocks terminal complement activation; therefore, patients may have increased susceptibility to infections, especially with encapsulated bacteria, such as infections caused by Neisseria meningitidis but also Streptococcus pneumoniae, Haemophilus influenzae, and to a lesser extent, Neisseria gonorrhoeae. Administer vaccinations for the prevention of Streptococcus pneumoniae infection according to ACIP recommendations. Patients receiving ZILBRYSQ are at increased risk for infections due to these organisms, even if they develop antibodies following vaccination.
Pancreatitis and pancreatic cysts have been reported in patients treated with ZILBRYSQ. During the open-label extension studies, seven (3.3%) patients experienced pancreatic events, including 4 (1.9%) patients with pancreatitis and 3 (1.4%) with pancreatic cysts.
In the 3-month, double-blind Study 1, adverse reactions of increased lipase were reported in six (6.9%) patients treated with ZILBRYSQ compared to no patients on placebo, and adverse reactions of increased amylase were reported in four (4.7%) patients treated with ZILBRYSQ compared to one (1.1%) patient on placebo. Lipase levels exceeded three times the upper limit of normal in six (7%) patients after being started on ZILBRYSQ compared to no patients on placebo.
Patients should be informed of this risk before starting ZILBRYSQ. Obtain lipase and amylase levels at baseline before starting treatment with ZILBRYSQ. Discontinue ZILBRYSQ in patients with suspected pancreatitis and initiate appropriate management until pancreatitis is ruled out or has resolved.
Advise the patients and/or caregivers to read FDA-approved patient labeling (Medication Guide and Instructions for Use).
Advise patients of the risk of serious meningococcal infection [see WARNINGS AND PRECAUTIONS]. Inform patients of the need to complete or update meningococcal vaccinations at least 2 weeks prior to receiving the first dose of ZILBRYSQ or receive antibacterial drug prophylaxis if ZILBRYSQ 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 ZILBRYSQ therapy.
Inform patients that vaccination may not prevent serious meningococcal infection and to seek immediate medical attention if the following signs or symptoms occur:
Inform patients that they will be given a Patient Safety Card for ZILBRYSQ that they should carry with them at all times and for 2 months following treatment with ZILBRYSQ. This card describes symptoms which, if experienced, should prompt the patient to immediately seek medical evaluation.
ZILBRYSQ is available only through a restricted program called ZILBRYSQ REMS [see WARNINGS AND PRECAUTIONS].
Inform the patient of the following notable requirements:
Counsel patients of the increased risk of infections, particularly those due to encapsulated bacteria, especially Neisseria species. Advise patients of the need for vaccination against meningococcal infections according to current medical guidelines [see WARNINGS AND PRECAUTIONS]. Counsel patients about gonorrhea prevention and advise regular testing for patients at risk. Advise patients to report any new signs and symptoms of infection.
Inform patients that pancreatitis and pancreatic cysts have been reported in patients taking ZILBRYSQ. Inform patients that persistent abdominal pain, sometimes severe or radiating to the back, which may or may not be accompanied by vomiting, is the hallmark symptom of acute pancreatitis. Instruct patients to contact their healthcare provider if these symptoms occur to determine if they should discontinue ZILBRYSQ [see WARNINGS AND PRECAUTIONS].
Provide training to patients and caregivers on proper subcutaneous injection technique.
Instruct patients to inject the full dose of ZILBRYSQ [see Medication Guide and Instructions for Use].
Instruct patients or caregivers in the technique of needle and syringe disposal [see Instructions for Use].
Remind patients if they forget to take their dose of ZILBRYSQ to inject their dose as soon as they remember. They should then take their next dose at the appropriate scheduled time. They should not take more than 1 dose per day.
Studies to assess the carcinogenic potential of zilucoplan have not been conducted.
Zilucoplan was not genotoxic in in vitro (Ames and chromosomal aberration) and in vivo (rat micronucleus) assays.
Subcutaneous administration of zilucoplan (0, 1, 2, or 4 mg/kg/day) to male monkeys resulted in testicular germ cell depletion/degeneration at all doses at the end of the 13Âweek dosing period and after the 8-week recovery period, indicating lack of reversibility. A no-effect dose for testicular germ cell degeneration was not identified. At the lowest dose tested, plasma exposures (AUC) were similar to that in humans at the maximum recommended human dose of 32.4 mg/day.
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.
Animal Data
Subcutaneous administration of zilucoplan (0, 1, 2, or 4 mg/kg/day) to pregnant monkeys throughout gestation resulted in an increase in embryofetal death at all doses, in the absence of maternal toxicity. A no effect dose for adverse developmental effects in monkeys was not identified. The lowest dose tested was associated with maternal exposures (AUC) similar to that in humans at the maximum recommended human dose of 32.4 mg/day.
Data from an ex vivo human placental transfer model demonstrated transfer of zilucoplan into the fetal compartment at a rate of 0.5% at a steady state plasma concentration of 10 μg/mL zilucoplan, which corresponds to a therapeutic dose of 0.3 mg/kg. The clinical significance of these data in human pregnancies is unknown.
There are no data on the presence of zilucoplan in human milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for ZILBRYSQ and any potential adverse effects on the breastfed infant from ZILBRYSQ or from the underlying maternal condition.
Safety and effectiveness in pediatric patients have not been established.
Clinical studies of ZILBRYSQ did not include sufficient numbers of patients aged 65 and older to determine whether they respond differently from younger adult patients.
No Information provided
ZILBRYSQ is contraindicated for initiation in patients with unresolved serious Neisseria meningitidis infection [see WARNINGS AND PRECAUTIONS].
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.
In a placebo-controlled Phase 2 study, patients received zilucoplan 0.3 mg/kg or 0.1 mg/kg for 12 weeks. There was a dose dependent inhibition of complement. Complement inhibition as represented by inhibition of sheep red blood cell (sRBC) lysis was 89.1% within 3 hours after the first administration and 94.9% after the 12-week treatment period in patients receiving ZILBRYSQ 0.3 mg/kg.
In Study 1 [see CLINICAL STUDIES], complement inhibition of 97.5% was observed by the end of the first week and sustained throughout the 12-week treatment period for gMG patients treated with the recommended dosage regimen of ZILBRYSQ.
At a dose two times the maximum approved recommended dose, ZILBRYSQ does not cause clinically significant QTc interval prolongation.
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.
Following single and multiple daily subcutaneous administration of ZILBRYSQ 0.3 mg/kg in healthy subjects, zilucoplan reached peak plasma concentration generally between 3 to 6 hours post-dose. Following daily subcutaneous dosing of ZILBRYSQ 0.3 mg/kg for 14 days in healthy subjects, both the peak plasma concentration and exposure (AUCtau) increased by approximately 3-fold.
In Study 1 [see Clinical Studies], after daily repeated subcutaneous administration of ZILBRYSQ 0.3 mg/kg, plasma concentrations of zilucoplan were consistent, with steady state trough concentrations being reached by four weeks of treatment with ZILBRYSQ through twelve weeks.
The mean volume of distribution at steady state was 3.51 L in the population pharmacokinetics analysis for adult patients with gMG. Zilucoplan and its 2 major metabolites are highly bound to plasma proteins (>99%).
The mean plasma terminal half-life of zilucoplan was approximately 172 hours (7 to 8 days).
Metabolism
As a peptide, ZILBRYSQ is expected to be degraded into small peptides and amino acids via catabolic pathways.
In plasma, two major metabolites, RA103488 and RA102758, were detected. The metabolite RA103488, formed mainly because of cytochrome CYP450 4F2, has pharmacological activity similar to zilucoplan but is present at a much lower concentration compared to zilucoplan. The metabolite RA102758, formed by protease mediated degradation, is pharmacologically inactive. The AUCs of both metabolites were approximately 10% of the parent AUC. The contribution of RA103488 to pharmacological activity is therefore expected to be low.
Excretion
Excretion of zilucoplan and its metabolite in urine and feces was negligible (<1% of the dose).
A population pharmacokinetics analysis assessing the effects of age, sex, and race did not suggest any clinically significant impact of these covariates on zilucoplan exposures.
A dedicated clinical study compared the pharmacokinetics of a single subcutaneous dose of ZILBRYSQ 0.3 mg/kg in subjects with severe renal impairment (as defined by a creatinine clearance <30 mL/min estimated by Cockcroft-Gault formula) to that of matched healthy subjects with normal renal function. A decrease in zilucoplan exposure (AUC0-inf) of 13% was observed. This change in zilucoplan exposures is not expected to be clinically significant. No dose adjustment is required in patients with renal impairment.
A dedicated clinical study compared the pharmacokinetics of a single subcutaneous dose of ZILBRYSQ 0.3 mg/kg in subjects with moderate hepatic impairment (as indicated by a Child-Pugh category of moderate [score of 7 to 9]) to that of matched healthy subjects with normal hepatic function. A decrease in zilucoplan exposure (AUC0Âinf) of 24% was observed. This change in zilucoplan exposures is not expected to be clinically significant. No dose adjustment is required in patients with mild or moderate hepatic impairment. The pharmacokinetics of zilucoplan in patients with severe hepatic impairment have not been studied.
Clinical drug interaction studies have not been performed with zilucoplan.
In vitro studies have shown that zilucoplan is not a substrate of major cytochrome P450 (CYP) enzymes (1A2, 2B6, 2C8, 2C9, 2C19, 2D6, and 3A) or transporters (P-gp, BCRP, OATP1B1, and OATP1B3). Based on the results from in vitro drug interaction testing, clinically relevant interactions between zilucoplan and substrates of CYP enzymes (1A2, 2B6, 2C8, 2C9, 2C19, 2D6, 3A, and 4F), uridine diphosphoglucuronosyl transferases (UGTs; 1A1, 1A3, 1A4, 1A6, 1A9, 2B7, and 2B15), and transporters (P-gp, BCRP, OATP1B1, OATP1B3, OAT1, OAT3, MATE1, MATE2-K, OCT1, and OCT2) is unlikely.
As with all therapeutic peptides, there is a potential for immunogenicity. 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 incidence of anti-drug antibodies in other studies or of other products. The sensitivity of the assay is not known although ADA was detectable in the tested samples.
In up to 12 weeks of treatment in Study 1, 2.3% (2/86) of patients treated with ZILBRYSQ developed anti-drug antibodies (ADA). A total of 9.3% (8/86) of ZILBRYSQ treated patients developed anti-polyethylene glycol (anti-PEG) antibodies. Because of the low occurrence of anti-drug antibodies and anti-PEG antibodies, the available data are too limited to make definitive conclusions regarding immunogenicity and its effect on pharmacokinetics, pharmacodynamics, safety, or efficacy of ZILBRYSQ.
The efficacy of ZILBRYSQ for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-AChR antibody positive was established in a 12-week, multicenter, randomized, double-blind placebo-controlled study (Study 1; NCT04115293).
Study 1 enrolled patients who met the following criteria at screening:
A total of 174 patients were enrolled in Study 1 and were randomized 1:1 to receive either ZILBRYSQ 0.3 mg/kg (n=86) or placebo (n=88) once daily by subcutaneous injection. Baseline characteristics were similar between treatment groups. Patients had a mean age of 53.0 years and a mean time since diagnosis of 9 years. Fifty-seven percent of the patients were female, 74% were White, 12% were Asian, 8% were Black, 1% were American Indian or Alaska Native, and 6% did not have race reported. The mean baseline MG-ADL total score was 10.6 (range 6 to 19).
At baseline, approximately 85% of patients in each group received cholinesterase inhibitors, 63% received steroids, and 51% received NSISTs, at stable doses.
The primary efficacy endpoint was a comparison of the change from baseline between treatment groups in the Myasthenia Gravis-Specific Activities of Daily Living scale (MGÂADL) total score after twelve weeks of treatment. The MG-ADL assesses the impact of gMG on daily functions of 8 signs or symptoms that are typically affected in gMG. Each item is assessed on a 4-point scale where a score of 0 represents normal function and a score of 3 represents loss of ability to perform that function. A total score ranges from 0 to 24, with the higher scores indicating more impairment.
The efficacy of ZILBRYSQ was also measured using the Quantitative Myasthenia Gravis (QMG) total score which is a 13-item categorical grading system that assesses muscle weakness. Each item is assessed on a 4-point scale where a score of 0 represents no weakness and a score of 3 represents severe weakness. A total possible score ranges from 0 to 39, where higher scores indicate more severe impairment.
Other secondary endpoints included the proportion of patients with improvements of at least 3 and 5 points in the MG-ADL total score and QMG total score, respectively, at week 12 without rescue therapy.
At week 12, treatment with ZILBRYSQ demonstrated a statistically significant improvement from baseline compared to placebo for MG-ADL total score and QMG total score (Table 3; Figure 1).
Table 3: Change from Baseline in MG-ADL and QMG Total Scores at Week 12 in Adult Patients with gMG who are Anti-AChR Antibody Positive (Study 1)
| Efficacy Endpoints: LS Mean (95% CI) | ZILBRYSQ (n = 86) |
Placebo (n = 88) |
ZILBRYSQ change LS mean difference vs. placebo (95% CI) | p-value* |
| MG-ADL Total Score | -4.39 (-5.28, -3.50) |
-2.30 (-3.17, -1.43) |
-2.09 (-3.24, -0.95) |
< 0.001 |
| QMG Total Score | -6.19 (-7.29, -5.08) |
-3.25 (-4.32, -2.17) |
-2.94 (-4.39, -1.49) |
< 0.001 |
| Abbreviations: CI = confidence interval; MG-ADL, myasthenia gravis activities of daily living scale; QMG, quantitative myasthenia gravis; LS = least square | ||||
Figure 1: Change from Baseline in MG-ADL Total Score (A) and QMG Total Score (B) through Week 12
 |
The proportion of MG-ADL responders with at least a 3-point improvement at week 12 was greater for ZILBRYSQ (73.1%) compared to placebo (46.1%) (p<0.001). The proportion of QMG responders with at least a 5-point improvement was also greater for ZILBRYSQ (58%) compared to placebo (33%) (p = 0.0012). The proportion of clinical responders at higher response thresholds was consistently greater for ZILBRYSQ compared to placebo.
ZILBRYSQ®
(ZIL-brisk)
(zilucoplan) injection, for subcutaneous use
What is the most important information I should know about ZILBRYSQ?
ZILBRYSQ is a medicine that affects part of your immune system. ZILBRYSQ may lower the ability of your immune system to fight infections.
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 2 months after your last ZILBRYSQ dose. Your risk of meningococcal infection may continue for several weeks after your last dose of ZILBRYSQ. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly.
ZILBRYSQ is only available through a program called the ZILBRYSQ Risk Evaluation and Mitigation Strategy (REMS). Before you can receive ZILBRYSQ, your healthcare provider must:
ZILBRYSQ may also increase the risk of other types of serious infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Haemophilus influenzae, and Neisseria gonorrhoeae.
Call your healthcare provider right away if you have new signs or symptoms of infection.
What is ZILBRYSQ?
Who should not use ZILBRYSQ?
Before you use ZILBRYSQ, tell your healthcare provider about all of your medical conditions, including if you:
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
Know the medicines you take and the vaccines you receive. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.
How should I use ZILBRYSQ?
See the detailed Instructions for Use that comes with your ZILBRYSQ for information on how to prepare and inject a dose of ZILBRYSQ, and how to properly throw away (dispose of) used ZILBRYSQ prefilled syringes.
If you miss your ZILBRYSQ dose, inject a dose as soon as possible. Then, inject your next dose at your regular scheduled time. Do not inject more than 1 dose each day. Call your healthcare provider if you are not sure what to do.
What are the possible side effects of ZILBRYSQ?
ZILBRYSQ may cause serious side effects, including:
The most common side effects of ZILBRYSQ include:
Tell your healthcare provider about any side effect that bothers you or that does not go away.
These are not all the possible side effects of ZILBRYSQ.
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
How should I store ZILBRYSQ?
Keep ZILBRYSQ prefilled syringes and all medicines out of the reach of children.
General information about the safe and effective use of ZILBRYSQ.
Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. Do not use ZILBRYSQ for a condition for which it was not prescribed. Do not give ZILBRYSQ to other people, even if they have the same symptoms that you have. It may harm them. You can ask your pharmacist or healthcare provider for information about ZILBRYSQ that is written for health professionals.
What are the ingredients in ZILBRYSQ?
Active ingredient: zilucoplan
Inactive ingredients: dibasic sodium phosphate, anhydrous, monobasic sodium phosphate, monohydrate, sodium chloride, and water for injection.
INSTRUCTIONS FOR USE
ZILBRYSQ®
(ZIL-brisk)(zilucoplan)injection, for subcutaneous useSingle-Dose Prefilled Syringe
This Instructions for Use contains information on how to inject ZILBRYSQ.
Understanding Your ZILBRYSQ Prefilled Syringe
Read this Instructions for Use before using the ZILBRYSQ prefilled syringe. This information does not take the place of talking to your healthcare provider about your medical condition or treatment. See the Medication Guide that comes with ZILBRYSQ for important information.
Your healthcare provider should show you how to prepare and inject ZILBRYSQ prefilled syringe properly before you use it for the first time. Do not inject yourself or someone else until you have been shown how to inject ZILBRYSQ correctly.
Keep this Instructions for Use and refer to it as needed until you have used all of the ZILBRYSQ prefilled syringes in the packaging.
For general questions or help, please call your healthcare provider or UCBCares at 1-844-599-CARE (2273).
How Should I Store ZILBRYSQ Prefilled Syringes?
Important Information You Need to Know Before You Inject ZILBRYSQ
ZILBRYSQ Prefilled Syringe Guide to Parts (Figure A):
Figure A
 |
Preparing for Injection
Step 1: Take out the ZILBRYSQ prefilled syringe
If the ZILBRYSQ prefilled syringes are stored in the refrigerator:
If the ZILBRYSQ prefilled syringes are stored at room temperature:
Figure B.1 AND Figure B.2
 |
Step 2: Inspect the ZILBRYSQ prefilled syringe
Figure C
 |
Step 3: Gather supplies
Wash your hands with soap and water and dry them with a clean towel. Gather the following supplies on a clean, flat surface (Figure D):
Figure D
 |
Step 4: Choose your injection site
Choose an injection site from the following areas (Figures E.1 and E.2):
Figure E.1 – abdomen and thighs
 |
Figure E.2 – upper arms
 |
Choose a different site each time you give yourself an injection. If you want to use the same injection site, make sure it is at least 1-inch from a spot you used before.
Do not inject ZILBRYSQ into an area that is red, tender, bruised, swollen, hard or that has scars or stretch marks.
Step 5: Clean your injection site
Clean the injection site using an alcohol wipe (Figure F).
Let the skin dry for 10 seconds before injecting.
Do not touch the injection site again before giving your injection.
Figure F
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Injecting the Medicine
Step 6: Remove the needle cap
Hold the body of the ZILBRYSQ prefilled syringe with one hand and pull the needle cap straight off with your other hand (Figure G).
Figure G
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Throw away (discard) the needle cap into your household trash or a sharps disposal container.
Do not touch the needle or let it touch anything.
Do not recap the needle at any time to avoid injury.
Do not try to remove any air bubbles from the ZILBRYSQ prefilled syringe. Air bubbles will not affect your dose and will not harm you. This is normal. You can continue to take your injection.
Step 7: Pinch your injection site
Use your other hand to pinch the area of cleaned skin and hold it firmly (Figure H).
Figure H
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Step 8: Insert the needle
Insert the entire needle into the pinched skin at a 45° to 90° angle. When the needle is fully inserted, hold the ZILBRYSQ prefilled syringe in place (Figure I).
Do not pull back on the plunger head at any time because this could break the prefilled syringe.
Do not touch the needle guard activation clips.
Figure I
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Step 9: Release the pinched skin
When the needle is fully inserted, hold the ZILBRYSQ prefilled syringe in place and release the pinched skin (Figure J).
Figure J
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Do not reinsert the needle into the skin if the needle is pulled out when releasing the skin. If this happens, safely throw away (dispose of) the syringe in a sharps disposal container and get a new ZILBRYSQ prefilled syringe to give the injection.
Step 10: Inject the medicine
Push the plunger head all the way down while holding onto the finger grip to inject all the medicine (Figure K).
Figure K
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Step 11: Release the plunger head
Slowly release the plunger head by lifting your thumb. After a complete injection, the needle guard will cover the needle and you may hear a click (Figure L).
Figure L
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Step 12: Throw away (dispose of) the used ZILBRYSQ prefilled syringe
Throw away (dispose of) the used ZILBRYSQ prefilled syringe into a sharps disposal container (Figure M) right away.
Do not throw away (dispose of) the ZILBRYSQ prefilled syringe in your household trash. If you do not have an FDA-cleared sharps disposal container, you may use a household container that is:
Do not throw away (dispose of) your used sharps disposal container in your household trash unless your community guidelines permit this.
Do not recycle your used sharps disposal container. Always keep the sharps disposal container out of the reach of children.
Figure M
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Note: When your sharps disposal container is almost full, you will need to follow your community guidelines for the right way to dispose of your sharps disposal container. There may be state or local laws about how you should throw away your used needles and syringes. For more information about safe sharps disposal, and for specific information about sharps disposal in the state you live in, go to the FDA’s website at: https://www.fda.gov/safesharpsdisposal.
Step 13: Examine the injection site
Press a cotton ball or gauze pad over the injection site and hold it for 10 seconds (Figure N).
Do not rub the injection site. You may have slight bleeding. This is normal. Apply an adhesive bandage if needed (Figure O).
Figure N AND Figure O
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The injection is complete.
For more information or help, contact your healthcare provider.
If you have any questions, call UCBCares at 1-844-599-CARE (2273) or visitwww.AskUCBCares.com.
This Instructions for Use has been approved by the U.S. Food and Drug Administration.
