ZILBRYSQ™ (zilucoplan injection) Now Approved for Adults with Generalized Myasthenia Gravis (gMG) in Canada

• ZILBRYSQ^TM (zilucoplan injection) is the first and only self-administered C5 complement inhibitor indicated for the treatment of generalized Myasthenia Gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive*.¹
• Health Canada's approval is supported by the pivotal Phase III RAISE study in gMG in adult patients which demonstrated that treatment with ZILBRYSQ resulted in statistically significant and clinically meaningful improvements in gMG-specific efficacy outcomes.²
• The approval of ZILBRYSQ for adults with anti-acetylcholine receptor (AChR) antibody positive gMG demonstrates UCB's commitment to advancing science in severe neurological and autoimmune diseases, addressing the gMG community's unmet needs and improving clinical outcomes.

OAKVILLE, ON, July 17, 2024 /CNW/ - UCB Canada Inc. is pleased to announce that ZILBRYSQ^TM (zilucoplan injection) received authorization from Health Canada on July 11, 2024 for the treatment of generalized Myasthenia Gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.* gMG is a rare, chronic autoimmune neuromuscular condition that causes skeletal muscle weakness, affecting the muscles of the eyes, face, neck, arms, legs, and throat. The onset of symptoms is usually quick and there is no cure.³

ZILBRYSQ is a C5 inhibitor and inhibits complement-mediated damage to the neuromuscular junction through its targeted dual mechanism of action.² Patients will be able to administer the ZILBRYSQ injection at home using pre-filled syringes. Benefits of subcutaneous self-injection can include reduced traveling time to and from hospitals, decreased interference with work obligations, and increased independence. Unlike monoclonal antibody C5 inhibitors, as a peptide, ZILBRYSQ can be used concomitantly with intravenous immunoglobulin and plasma exchange, without the need for supplemental dosing.²

"Health Canada's approval of ZILBRYSQ marks a significant milestone for UCB Canada. As our first step in supporting the gMG community in Canada, this achievement represents a breakthrough in a new therapeutic area for our company," says Rodrigo Reis, Country Lead, UCB Canada. "Looking ahead, we are hopeful that we will have two approved therapeutic solutions for gMG patients by the end of 2024. Our focus remains on pioneering scientific solutions that elevate the standard of care, enhancing the lives of those affected by gMG and other rare diseases."

Health Canada approval of ZILBRYSQ is based on UCB's RAISE study which demonstrated that zilucoplan delivered rapid, consistent, statistically significant and clinically meaningful benefits in different patient-and-clinician-reported outcomes – Myasthenia Gravis-Activities of Daily Living (MG-ADL) score, Quantitative Myasthenia Gravis (QMG) score, Myasthenia Gravis Composite (MGC) score and Myasthenia Gravis Quality of Life 15-item scale (MG-QoL15r)** – at week 12 in a broad population of mild to severe adult patients with AChR antibody positive gMG.²

"Until now, Canadians living with gMG have only had access to C5 therapy intravenously, which can be time-consuming and burdensome for the health care system," explained Dr. Angela Genge, Medical Director, Clinical Research Unit (CRU), Montreal Neurological Institute and international leader in clinical trial design and development for rare neurological conditions. "Health Canada's approval of the first once-daily and only self-administered C5 complement inhibitor that provides significant and clinically meaningful improvements addresses a great unmet need by providing people living with this often-debilitating rare disease with choice and flexibility." 

"Generalized myasthenia gravis can profoundly impact a person's physical, emotional, social, and financial well-being. The unpredictable and fluctuating symptoms can be debilitating, significantly affecting patients' day-to-day lives," said Stacey Lintern, Chief Executive Officer, Muscular Dystrophy Canada. "The gMG community in Canada has long awaited new treatment options that extend beyond symptom-management and offer the potential to improve outcomes and reduce burden. Today's announcement brings new hope for people with gMG, introducing a treatment option that promises to enhance the quality of life for many Canadians. This progress marks a significant step forward in the ongoing need for improved and accessible treatment options for gMG, offering renewed optimism for patients and their families."

UCB Canada anticipates supply availability of ZILBRYSQ in Q4 2024.

About Generalized Myasthenia Gravis
Generalized Myasthenia Gravis (gMG) is a rare chronic, autoimmune neuromuscular disease characterized by muscle weakness that worsens after periods of activity and improves after periods of rest. gMG can affect people of all races, ages and genders but is not thought to be directly inherited, nor is it contagious.^4,5

Approximately 32 out of every 100,000 people are living with gMG⁶ and it affects each person differently. Common symptoms include chronic fatigue and weakness in different muscle groups, that can impact daily activities – such as tooth-brushing, hair-combing, speaking (dysarthria) or swallowing (dysphagia) – and quality of life. In addition, symptom severity can increase unexpectedly, and might lead to hospitalization.⁵

gMG can have a significant economic impact on those living with it, with loss of income and lack of funds for emergency care cited as particularly challenging.⁷ The condition is associated with substantial healthcare costs and resource use, with hospitalization being the most significant factor.⁸

About the RAISE Study
The RAISE study (NCT04115293) was a multi-centre, Phase III, randomized, double-blind, placebo-controlled study to confirm the efficacy, safety, and tolerability of zilucoplan in adult patients with anti-acetylcholine receptor (AChR) antibody positive gMG. Patients were randomized in a 1:1 ratio to receive once daily subcutaneous injection of 0.3 mg/kg zilucoplan or placebo for 12 weeks.

The primary endpoint for the RAISE study was change from baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score. Secondary endpoints included change from baseline in the Quantitative Myasthenia Gravis (QMG) score, the Myasthenia Gravis Composite (MGC) and the Myasthenia Gravis Quality of Life 15 revised (MG-QoL15r) score from baseline to Week 12, time to first rescue therapy, the proportion of patients with minimal symptom expression (MSE) (defined as MG-ADL of 0 or 1 without rescue therapy), the proportion with a ≥3-point reduction in MG-ADL without rescue therapy and the proportion with a ≥5-point reduction in QMG without rescue therapy, all measured at Week 12. Safety was assessed by the incidence of treatment emergent adverse events (TEAEs). Patients who completed the RAISE trial had the possibility to enter the open-label extension study, RAISE-XT (NCT04225871).²

More information about ZILBRYSQ can be found by accessing the product monograph.

About UCB Canada Inc.
Inspired by patients and driven by science, UCB Canada Inc. is a biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe autoimmune and central nervous system diseases. For more information, please consult https://www.ucb-canada.ca/.

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 approximately 8,400 people in nearly 40 countries, the company generated revenue of €5.3 billion in 2020. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news. 

* Patients continued to receive standard therapy throughout the pivotal trial.
**The threshold for clinical meaningfulness for MG-QoL 15r has not be established.

References

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¹ ZILBRYSQ Product Monograph, 2024.

² Howard JF Jr et al. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase  study. Lancet Neurol. 2023;22:395-406.

³ Cleveland Clinic. Myasthenia Gravis (MG), Myasthenia Gravis: What It Is, Causes, Symptoms & Treatment (clevelandclinic.org). Accessed May 2024.

⁴ National Institute of Neurological Disorders and Stroke. What is Myasthenia Gravis. www.ninds.nih.gov/health-information/disorders/myasthenia-gravis. Accessed May 2024.

⁵ Muscular Dystrophy Canada. Myasthenia Gravis: Know All About It. ALE19SO021024_PSPMGDiseasesBrochure_E7_v2.pdf (muscle.ca). Accessed May 2024.

⁶ Bubuioc, A. M., Kudebayeva, A., Turuspekova, S., Lisnic, V., & Leone, M. A. (2021). The epidemiology of myasthenia gravis. Journal of medicine and life, 14(1), 7–16. https://doi.org/10.25122/jml-2020-0145. 

⁷ Hughes Tom et al. The economic burden of individuals living with generalized myasthenia gravis and facing social determinants of health challenges. Front Public Health. 2023 Sep 12:11:1247931. https://pubmed.ncbi.nlm.nih.gov/37766748/. Accessed June 2024.

⁸ Zhdanava, M et al. Economic burden of generalized myasthenia gravis (MG) in the United States and the impact of common comorbidities and acute MG-events. Current Medical Research and Opinion, 1–9. www.tandfonline.com/doi/full/10.1080/03007995.2024.2353381. Accessed June 2024.

SOURCE UCB Canada Inc.

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