Zilucoplan Autoinjector Matches Bioequivalence of Pre-Filled Syringe in Myasthenia Gravis

New data presented at the 2026 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting showed that zilucoplan delivered through a pre-filled autoinjector is bioequivalent to the currently approved pre-filled syringe and was effectively and safely self-administered by patients with generalized myasthenia gravis (gMG). The findings support a pre-filled pen presentation of zilucoplan that UCB, the drug's developer, is already advancing through European regulatory review.^1,2

Bioequivalence in healthy volunteers

The phase 1 DV0012 study (NCT06511076) randomized 14 healthy adults 1:1 to a single subcutaneous injection with the zilucoplan autoinjector followed by the pre-filled syringe, or the reverse sequence, to compare pharmacokinetics between the 2 delivery devices. Geometric least-squares mean ratios (90% CI) comparing the autoinjector with the syringe were 0.98 (0.95-1.01) for area under the concentration-time curve (AUC), 0.98 (0.95-1.01) for AUC up to the last quantifiable concentration, and 1.00 (0.96-1.04) for maximum observed concentration, results the investigators said established bioequivalence between the 2 presentations.¹

Self-administration in patients with gMG

The phase 3b DV0013 study (NCT06471361) enrolled 31 patients with gMG who self-administered zilucoplan once daily using the autoinjector. Complete dose delivery, confirmed by investigators, was achieved in 99.8% (95% CI, 98.8-100) of injections through day 14, the study's primary objective. Treatment-emergent adverse events (TEAEs) occurred in 22.6% of patients (7 of 31), all mild and nonserious, and median satisfaction with self-injection on the Self-Injection Assessment Questionnaire was 8.9 (range, 6-10) at day 14.¹

“Zilucoplan bioequivalence was established between [the autoinjector] and [the pre-filled syringe]. Self-administration with the autoinjector achieved high complete dose delivery and self-injection satisfaction, and was well tolerated in patients with gMG, suggesting that [it] is effective for zilucoplan administration,” concluded Mary Petrulis, MD, assistant professor of neurology at the Ohio State University Wexner Medical Center, and colleagues.

A pre-filled pen advancing through EU review

The DV0012 and DV0013 data also support a variation UCB has filed with European regulators to add a pre-filled pen as a new device presentation for zilucoplan (Zilbrysq), which the European Medicines Agency's Committee for Medicinal Products for Human Use recommended for approval in March 2026. Zilucoplan, a complement component 5 inhibitor, is currently approved in the United States and European Union as a once-daily subcutaneous injection administered via pre-filled syringe for adults with anti-acetylcholine receptor antibody-positive gMG.^2,3

“The zilucoplan pre-filled pen may support confidence in daily treatment through simplified administration," Petrulis said of the device separately from the AANEM data.² Donatello Crocetta, MD, chief medical officer at UCB, said, “By simplifying daily administration, this new device supports confidence in self-management.”

Zilucoplan's approval rests on the phase 3 RAISE trial, in which the drug produced a placebo-corrected 2.09-point improvement in MG-ADL score at 12 weeks, alongside a QMG score improvement of 6.19 points versus 3.25 points with placebo; MG worsening occurred in 10.5% of zilucoplan-treated patients compared with 25.0% on placebo.⁴

Click here for more AANEM 2026 coverage.

REFERENCES
1. Petrulis M, Howard JF Jr, Hussain Y, Ilkowski J, Khatri B, Leite MI, Nowak R, Shah S, Vu T, Boroojerdi B, Brock M, Cleverly A, Delicha EM, Hajihosseini A, Hernandez MA. Bioequivalence and effectiveness of autoinjector-delivered, self-administered zilucoplan compared with pre-filled syringe. Presented at: 2026 AANEM Annual Meeting and MGFA Scientific Session; September 29-October 2, 2026; Orlando, FL.
2. UCB receives positive CHMP opinion for new ZILBRYSQ (zilucoplan) pre-filled pen in EU for adults living with generalized myasthenia gravis. News release. Published March 26, 2026. Accessed September 28, 2026. https://www.ucb.com/newsroom/press-releases/article/ucb-receives-positive-chmp-opinion-for-new-zilbrysqrv-zilucoplan-pre-filled-pen-in-eu-for-adults-living-with-generalized-myasthenia-gravis
3. UCB announces U.S. FDA approval of ZILBRYSQ (zilucoplan) for the treatment of adults with generalized myasthenia gravis. News release. Published October 17, 2023. Accessed September 28, 2026. https://www.ucb.com/newsroom/press-releases/article/ucb-announces-us-fda-approval-of-zilbrysqr-zilucoplan-for-the-treatment-of-adults-with-generalized-myasthenia-gravis
4. Howard JF Jr, et al. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet Neurol. Published online April 13, 2023. doi:10.1016/S1474-4422(23)00080-7