SAFETY
Examine safety profiles from RAISE and RAISE-XT
RAISE: safety and tolerability were demonstrated in a Phase 3 pivotal study1,2
Adverse reactions in ≥5% of patients treated with ZILBRYSQ and more frequently than in patients who received placebo.1
%
%
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.1
Immunize patients with meningococcal vaccines at least 2 weeks before administering the first dose of ZILBRYSQ.1
RAISE-XT primary endpoint: safety and tolerability3,4
ZILBRYSQ was generally well-tolerated with a favorable safety profile at a median exposure of 1.8 years (range: 0.11–5.1 years).4
- TEAEs seen in the RAISE-XT study were similar to those seen in the RAISE study. No new safety concerns were raised
- 9.5% of patients (n=19) discontinued ZILBRYSQ by Week E84 due to TEAEs
In RAISE-XT, 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 due to morphea.1
Most common treatment-emergent adverse events (TEAEs)4
(N=200)
Includes all deaths.
No deaths were considered treatment related. TEAEs leading to death included cardiac arrest (n=2) and accidental head injury (n=1) in the zilucoplan 0.3 mg/kg / 0.3 mg/kg group, and death from an unknown cause (n=1) in the placebo / zilucoplan 0.3 mg/kg group.
Only most common TEAEs, occurring in ≥15% of patients overall, are reported.
Data cut-off: 11 May 2023.
COVID-19=coronavirus disease 2019; IMP=investigational medicinal product; TEAE=treatment-emergent adverse event; URTI=upper respiratory tract infection.
Additional ZILBRYSQ warnings and precautions1
Other infections
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 and Haemophilus influenzae type b (Hib) infections according to ACIP guidelines. Persons receiving ZILBRYSQ are at increased risk for infections due to these bacteria, even after vaccination.
Pancreatitis and other pancreatic conditions
Pancreatitis and pancreatic cysts have been reported in patients treated with ZILBRYSQ.
RAISE-XT
n (%)
During the open-label extension studies, 7 (3.3%) patients experienced pancreatic events, including 4 (1.9%) patients with pancreatitis and 3 (1.4%) with pancreatic cysts.
Patients should be informed of the risk of pancreatitis and other pancreatic conditions before starting ZILBRYSQ.
Discontinue ZILBRYSQ in patients with suspected pancreatitis and initiate appropriate management until pancreatitis is ruled out or has resolved.
RAISE
n (%)
n (%)
In the 3-month, double-blind Study 1, adverse reactions of increased lipase were reported in 6 (6.9%) patients treated with ZILBRYSQ compared to no patients on placebo, and adverse reactions of increased amylase were reported in 4 (4.7%) patients treated with ZILBRYSQ compared to 1 (1.1%) patient on placebo. Lipase levels exceeded three times the upper limit of normal in 6 (7%) patients after being started on ZILBRYSQ compared to no patients on placebo.
Obtain lipase and amylase levels at baseline before starting treatment with ZILBRYSQ.
References:
- ZILBRYSQ [Prescribing Information]. Smyrna, GA: UCB, Inc.
- Vu T, Genge A, Hussain Y, et al; on behalf of the RAISE investigators. Efficacy and safety of zilucoplan in myasthenia gravis: responder analysis from the randomized phase 3 RAISE trial. Poster presented at: American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting; September 21-24, 2022; Nashville, TN. Poster 200.
- Howard JF Jr, Bresch S, Farmakidis C, et al. Long-term safety and efficacy of zilucoplan in patients with generalized myasthenia gravis: interim analysis of the RAISE-XT open-label extension study. Ther Adv Neurol Disord. 2024;17(3):1–16. doi:10.1177/17562864241243186
- Leite MI, Bresch S, Hewamadduma C, et al; on behalf of the RAISE-XT study team. Long-term zilucoplan in generalised myasthenia gravis: 96-week follow-up interim analysis of RAISE-XT. Presented at European Academy of Neurology 2024 Annual Meeting; April 13-18, 2024; Helsinki, Finland. Presentation EPR-254.
