UCB receives positive CHMP opinion for new ZILBRYSQ[®]▼ (zilucoplan) pre-filled pen in EU for adults living with generalized myasthenia gravis

Brussels (Belgium) 26 March 2026, 07:00 (CET) – UCB (Euronext Brussels: UCB), a global biopharmaceutical company, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending a variation to the terms of the marketing authorization for ZILBRYSQ^® (zilucoplan), introducing a new additional device presentation via a pre-filled pen as an add-on to standard therapy for the treatment of adults living with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.^1,3

Zilucoplan pre-filled pen is a self-administration device designed to automatically inject an accurate and defined dose of zilucoplan triggered by push-on-skin activation.^1,4 It is the first once-daily, subcutaneous C5 inhibitor for gMG in Europe commercially available as a pre-filled pen and provides an additional application option for adults with anti-AChR antibody-positive gMG.¹ Up to now, zilucoplan was approved in Europe to be self-administered subcutaneously by patients or caregivers using a pre-filled syringe injection.¹

“The approval of the zilucoplan pre-filled pen reflects our commitment not only to advancing the science in generalized myasthenia gravis, but also to innovating how treatments are delivered and experienced,” said Donatello Crocetta, MD, Chief Medical Officer at UCB. “By simplifying daily administration, this new device is designed to support confidence in self-management while delivering the same trusted therapy supported by the established clinical evidence of zilucoplan. Ultimately, it reinforces our focus on translating scientific innovation into meaningful improvements in patients’ daily lives.”

The pre-filled pen offers a portable solution that is designed to fit into patients’ daily routines while delivering high self‑injection satisfaction,^1,4 empowering patients to take control of their treatment journey at home.⁵ As the needle in the pre-filled pen is not visible, it has the added benefit of potentially helping patients overcome needle-related concerns.^1,6 

This advancement significantly supports UCB’s environmental goals as it features an optimized product and packaging design that achieves up to a 50% reduction in CO₂ emissions compared to other production methods.^2,7

“When developing new delivery options, it is essential to generate robust clinical evidence that evaluates both the performance of the medicine and how it is used in real-world settings,” said Dr. Mary Petrulis, Neurologist at The Ohio State University Wexner Medical Center. “For people living with generalized myasthenia gravis, the zilucoplan pre-filled pen may support confidence in daily treatment through a simplified administration approach. Its design allows patients to administer therapy as part of their routine, while preserving the established clinical profile of the therapy.”

Supporting data

Data from two clinical studies* DV0012 and DV0013 demonstrated bioequivalence between zilucoplan pre-filled pen and pre-filled syringe and successful self-administration with the pre-filled pen.⁴ UCB will present these data at this year’s American Academy of Neurology (AAN) meeting, Chicago, Illinois, April 18-22, 2026. The DV0012 study (NCT06511076) established zilucoplan bioequivalence in healthy volunteers between the pre-filled pen and pre-filled syringe, thereby confirming that the pre-filled pen may be an effective alternative for zilucopan administration.^1,4 In a separate study in adults with gMG, results from the DV0013 study (NCT06471361) showed that self-administration with the pre-filled pen achieved complete dose delivery in 99.8% of administrations, and was generally well tolerated.⁴ Additionally, at day 14 median (range) 'satisfaction with self-injection' score, measured as part of the Self-Injection Assessment Questionnaire, results showed a score of 8.9 (6-10).⁴

About zilucoplan

Zilucoplan is a once-daily SC, self-administered peptide inhibitor of complement component 5 (C5 inhibitor). As a C5 inhibitor, zilucoplan inhibits complement-mediated damage to the neuromuscular junction through its targeted dual mechanism of action.^1,5 Zilucoplan received European Commission approval in December 2023 as an add-on to standard therapy for the treatment of adults with anti-AChR antibody-positive gMG.² The approval was supported by safety and efficacy data evaluated in a 12-week multicenter, randomized, double-blind placebo-controlled study MG0010 (RAISE) and the open-label extension study MG0011 (RAISE XT). Zilucoplan met the primary endpoint in RAISE which was change from baseline to week 12 in MG-ADL total score versus placebo.¹

About generalized Myasthenia Gravis (gMG)

gMG is a rare autoimmune disease with a global prevalence of 100–350 cases per every 1 million people.⁸ People living with gMG can experience a variety of symptoms, including severe muscular weakness that can result in double vision, drooping eyelids, difficulty with swallowing, chewing and talking, as well as life-threatening weakness of the muscles of respiration.^9,10

*DV0012 was a Phase 1, open-label, single-center, two-period crossover study in healthy adult volunteers randomized 1:1 to one of two treatment sequences: single injection with ZLP-AI (auto-injector) then ZLP-PFS (pre-filled syringe), or ZLP-PFS then ZLP-AI. Primary PK parameters estimated were area under the curve (AUC), AUC up to last quantifiable concentration (AUC0–t) and maximum observed concentration (C_max). DV0013 was a Phase 3b, open-label, multicenter study, in patients with gMG either participating in RAISE-XT (NCT04225871) or receiving commercially administered zilucoplan; patients self-administered once-daily ZLP-AI. Primary objective was the effective ZLP-AI self-administration through Day 14, defined as investigator confirmed complete dose delivery. Satisfaction with self-injection, measured by the Self-Injection Assessment Questionnaire (SIAQ, domain scores 0–10) and safety were also assessed.⁴

ZILBRYSQ^®▼ (zilucoplan) EU/EEA** Important Safety Information¹

▼This medicinal product is subject to additional monitoring. This will allow quick identification of new

safety information. Healthcare professionals are asked to report any suspected adverse reactions.

Zilbrysq is indicated as an add-on to standard therapy for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR ) antibody positive in three approved doses: 16.6, 23 and 32.4 mg.

The most frequently reported adverse reactions were injection site reactions (injection site bruising (13.9%) and injection site pain (7.0%)) and upper respiratory tract infections (nasopharyngitis (5.2%), upper respiratory tract infection (3.5%) and sinusitis (3.5%)). The adverse reactions from the pooled placebo-controlled (n=115) and open-label extension (n=213) studies in gMG are as follows: Very common adverse reactions: (≥ 1/10): Upper respiratory tract infections and Injection site reactions; Common adverse reactions (≥ 1/100 to < 1/10): Diarrhea, Lipase increased, Amylase increased and Morphoea; Uncommon adverse reaction ((≥ 1/1000 to < 1/100) blood eosinophils increased.

Zilucoplan is contra-indicated in patients with hypersensitivity to the active substance or to any of the excipients, in patients who are not currently vaccinated against Neisseria meningitidis and in patients with unresolved Neisseria meningitidis infection. Due to its mechanism of action, the use of zilucoplan may increase the patient’s susceptibility to infections with Neisseria meningitidis. As a precautionary measure, all patients must be vaccinated against meningococcal infections, at least 2 weeks prior to the start of treatment. If treatment needs to start less than 2 weeks after vaccination against meningococcal infections, the patient must receive appropriate prophylactic antibiotic treatment until 2 weeks after the first vaccination dose. Meningococcal vaccines reduce but do not completely eliminate the risk of meningococcal infections. Vaccines against serogroups A, C, Y, W, and where available, serogroup B, are recommended for preventing the commonly pathogenic meningococcal serogroups. Vaccination and prophylactic antibiotic treatment should occur according to most current relevant guidelines. During treatment, patients should be monitored for signs and symptoms of meningococcal infection and evaluated immediately if infection is suspected. In case of a suspected meningococcal infection, appropriate measures such as treatment with antibiotics and discontinuation of treatment, should be taken until the meningococcal infection can be ruled out.

Patients should be instructed to seek immediate medical advice if signs or symptoms of meningococcal infections occur. Prescribers should be familiar with the educational materials for the management of meningococcal infections and provide a patient alert card and patient/carer guide to patients treated with zilucoplan. In addition to Neisseria meningitidis, patients treated with zilucoplan may also be susceptible to infections with other Neisseria species, such as gonococcal infections. Patients should be informed on the importance of gonorrhea prevention and treatment. Prior to initiating zilucoplan therapy, it is recommended that patients initiate immunizations according to current immunization guidelines.

Please consult the full product information in relation to other side effects, full safety profile and prescribing information SmPC.

 Date of last revision: 19 March 2026.

**EU/EEA means European Union/European Economic Area.

For further information, contact UCB:

Global Communications

Nick Francis

T: +44 7769 307745

nick.francis@ucb.com

Corporate Communications, Media Relations

Laurent Schots

T: +32.2.559.92.64

laurent.schots@ucb.com

Investor Relations

Antje Witte

T: +32.2.559.94.14

antje.witte@ucb.com

About UCB

UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With more than 9,000 people in approximately 40 countries, the company generated revenue of €7.7 billion in 2025. UCB is listed on Euronext Brussels (symbol: UCB). 

Forward-looking statements

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References

 

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