UCB Presents Latest Data from Generalized Myasthenia Gravis Portfolio at 9th Congress of the European Academy of Neurology (EAN) Meeting
Brussels, Belgium, 30th June 2023 – 18:30 PM CEST– UCB, a global biopharmaceutical company, today announced it will present results from across its portfolio in generalized myasthenia gravis (gMG) at the European Academy of Neurology (EAN) Meeting July 1-4, 2023.
Additional results from the MycarinG and RAISE studies1,2, and their open-label extension studies will be presented, investigating UCB’s rozanolixizumab, an SC-infused monoclonal antibody targeting the neonatal Fc receptor (FcRn)3,4 and zilucoplan, a self-administered, subcutaneous (SC) peptide inhibitor of complement component 5 (C5 inhibitor) in adults with gMG.5 These pivotal Phase 3 trials supported U.S., European, and Japanese regulatory filings of both rozanolixizumab and zilucoplan. Presentations will also focus on the importance of real-world data and its use in better understanding current patient experiences. In total, nine abstracts will be presented, including three oral presentations.
In June, rozanolixizumab was approved by the U.S. Food and Drug Administration (FDA), for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AchR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive,6 having been granted Priority Review for its Biologic License Application (BLA). In the U.S., under the FDAs non-proprietary naming of biological products guidance, the nonproprietary name for this medicine is rozanolixizumab-noli. It is an FDA-approved treatment for both anti-AChR and anti-MuSK antibody-positive gMG, the two most common subtypes of gMG. Rozanolixizumab is currently only approved in the U.S., and is under review by the European Medicines Agency (EMA) and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) for the treatment of adults with gMG. Responses from regulatory agencies to these submissions are expected by H1 2024.
Zilucoplan is currently under review by the U.S. FDA, EMA and the Japanese PMDA for the treatment of adult gMG patients who are anti-acetylcholine receptor (AChR) antibody positive. Responses from these regulatory agencies are expected from H2 2023 onwards. The safety and efficacy of zilucoplan have not been established and it is not currently approved for use in any indication by any regulatory authority worldwide.
These data further inform UCB’s innovative approach to evolving science into meaningful solutions that help improve outcomes and address unmet needs of people living with gMG.
Donatello Crocetta, Head of Global Rare Disease & Rare Medical, UCB, explained: “The additional results from the Phase 3 MycarinG and RAISE studies demonstrate UCB’s dedication to finding treatment options for patients with gMG and reinforce the depth and strength of our expanding rare disease pipeline and portfolio. Following on from our recent approval for rozanolixizumab in the U.S. for the treatment of adult patients who are anti-acetylcholine receptor or anti-muscle-specific tyrosine kinase antibody positive gMG, we’re very excited to be collaborating with the gMG community to extend knowledge about new medicines which could, in the future, play an important role in the treatment of this rare neuromuscular disease.”
Data from UCB's extensive work to better understand the burden of gMG in different European countries will also be presented in 4 posters. Additionally, through an immersive VR experience co-created with the members of the gMG community, UCB will enable HCPs to gain a deeper understanding of the daily challenges experienced by people living with gMG.
“Because of the high disease and treatment burden that gMG elicits along with the impact on a person’s daily life, it is important for us to contribute and improve our wealth of knowledge of this debilitating rare disease - especially relevant following Myasthenia Gravis Awareness Month by focusing - on patients and digital innovations, with the aim to address the needs of people living with gMG.” said Manuela Maronati, Head of Europe, Rare Disease, UCB. “As well as increasing our understanding of gMG, we hope to continue to work towards bringing new treatment options to patients. We are looking forward to hearing from the European Medicines Agency on their review of the Marketing Authorization Application for both rozanolixizumab and zilucoplan.”
UCB presentations during EAN 2023
About Generalized Myasthenia Gravis (gMG)
gMG is a rare disease with a global prevalence of 100–350 cases per every 1 million people.7 People living with gMG can experience a variety of symptoms, including severe muscular weakness that can result in life-threatening weakness of the muscles of respiration, double vision, drooping eyelids, and difficulty swallowing, chewing and talking.8,9
In gMG, pathogenic autoantibodies can impair synaptic transmission at the neuromuscular junction (NMJ) by targeting specific proteins on the post-synaptic membrane.3,10 This disrupts the ability of the nerves to stimulate the muscle and results in a weaker contraction. gMG can occur in any race, gender or age.11
Rozanolixizumab is a subcutaneous administered, humanized monoclonal antibody that specifically binds, with high affinity, to human neonatal Fc receptor (FcRn). It has been designed to block the interaction of FcRn and Immunoglobulin G (IgG), accelerating the catabolism of antibodies and reducing the concentration of pathogenic IgG autoantibodies.12
Outside of the U.S. rozanolixizumab is not approved for use in any indication by any other regulatory authority worldwide.
Important U.S. Safety Information for RYSTIGGO®
WARNINGS AND PRECAUTIONS
Infections: RYSTIGGO may increase the risk of infection. Delay RYSTIGGO administration in patients with an active infection until the infection is resolved. During treatment with RYSTIGGO, monitor for clinical signs and symptoms of infection. If serious infection occurs, administer appropriate treatment and consider withholding RYSTIGGO until the infection has resolved.
Immunization with vaccines during RYSTIGGO treatment has not been studied. The safety of immunization with live or live-attenuated vaccines and the response to immunization with any vaccine are unknown. Because RYSTIGGO causes a reduction in IgG levels, vaccination with live-attenuated or live vaccines is not recommended during treatment with RYSTIGGO. Evaluate the need to administer age-appropriate vaccines according to immunization guidelines before initiation of a new treatment cycle with RYSTIGGO.
Aseptic Meningitis: Serious adverse reactions of aseptic meningitis (also called drug-induced aseptic meningitis) have been reported in patients treated with RYSTIGGO. If symptoms consistent with aseptic meningitis develop, diagnostic workup and treatment should be initiated according to the standard of care.
Hypersensitivity Reactions: Hypersensitivity reactions, including angioedema and rash, were observed in patients treated with RYSTIGGO. Management of hypersensitivity reactions depends on the type and severity of the reaction. Monitor patients during treatment with RYSTIGGO and for 15 minutes after for clinical signs and symptoms of hypersensitivity reactions. If a reaction occurs, institute appropriate measures if needed.
In a placebo-controlled study, the most common adverse reactions (reported in at least 10% of RYSTIGGO-treated patients) were headache, infections, diarrhea, pyrexia, hypersensitivity reactions, and nausea. Serious infections were reported in 4% of patients treated with RYSTIGGO. Three fatal cases of pneumonia were identified, caused by COVID-19 infection in two patients and an unknown pathogen in one patient. Six cases of infections led to discontinuation of RYSTIGGO.
Full U.S. Prescribing Information is available at https://www.ucb-usa.com/RYSTIGGO-prescribing-information.pdf
Zilucoplan is a once-daily subcutaneous (SC), self-administered peptide inhibitor of complement component 5 (C5 inhibitor) under clinical development by UCB in gMG. As a C5 inhibitor, zilucoplan inhibits complement-mediated damage to the neuromuscular junction through its targeted dual mechanism of action.13 In 2019, the U.S. FDA granted orphan drug designation to zilucoplan for the treatment of myasthenia gravis.14 Orphan designation was granted in July 2022 by the European Commission to zilucoplan for the treatment of myasthenia gravis.15 The safety and efficacy of zilucoplan have not been established and it is not currently approved for use in any indication by any regulatory authority worldwide.
For further information, contact UCB:
T +44 7900 605 652
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 8 600 people in approximately 40 countries, the company generated revenue of € 5.8 billion in 2021. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news
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- Clinical Trials.gov. 'A Study to Test Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis'. https://clinicaltrials.gov/ct2/show/NCT03971422. Accessed June 2023.
- Clinical Trials.gov. 'Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis (RAISE)' https://clinicaltrials.gov/ct2/show/NCT04115293. Accessed June 2023.
- Smith B, et al. Generation and characterization of a high affinity anti-human FcRn antibody, rozanolixizumab, and the effects of different molecular formats on the reduction of plasma IgG concentration. MAbs. 2018;10:1111-30.
- Kiessling P, et al. The FcRn inhibitor rozanolixizumab reduces human serum IgG concentration: A randomized phase 1 study. Sci Transl Med. 2017;9(414:eaan1208).
- Howard J, et al. Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial. JAMA Neurol. 2020 May 1;77(5):582-92.
- RYSTIGGO® U.S. Prescribing Information
- Punga AR, et al. Epidemiology, diagnostics, and biomarkers of autoimmune neuromuscular junction disorders. Lancet Neurol. 2022;21(2):176-88.
- Myasthenia Gravis Foundation of America. Clinical Overview of MG. https://myasthenia.org/Professionals/Clinical-Overview-of-MG. Accessed June 2023.
- Hansen JS, et al. Mortality in myasthenia gravis: A nationwide population-based follow-up study in Denmark. Muscle Nerve. 2016;53: 73-7.
- Juel VC, Massey JM. Myasthenia gravis. Orphanet J Rare Dis. 2007;2:44.
- Myasthenia Gravis Foundation of America. MG Quick Facts. https://myasthenia.org/MG-Education/MG-Quick-Facts. Accessed June 2023.
- Wolfe G, et al. Myasthenia gravis activities of daily living profile. Neurology. 1992;52(7):1487-9.
- Howard J, et al. Efficacy and safety of zilucoplan in patients with generalised myasthenia gravis: A randomised, double-blind, placebo-controlled, Phase 3 study (RAISE). Lancet Neurol. 2023 May;22(5):395-406.
- US Food and Drug Administration. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=699319. Accessed June 2023.
- European Medicines Agency, EU/3/22/2650: Orphan designation for the treatment of myasthenia gravis https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2650. Accessed June 2023
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