ATLANTA — UCB (Euronext Brussels: UCB), a global biopharmaceutical company, today announced that it will be presenting results from across its portfolio in generalized myasthenia gravis (gMG) at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) annual meeting and the Myasthenia Gravis Association of America Scientific Session taking place November 1-4, 2023.
Additional results from the MycarinG and RAISE studies,1,2 and their open-label extension studies will be presented, investigating UCB’s rozanolixizumab-noli, a subcutaneously (SC)-injected monoclonal antibody targeting the neonatal Fc receptor (FcRn)3,4 and zilucoplan, a self-administered, subcutaneously injected peptide inhibitor of complement component 5 (C5 inhibitor) in adults with gMG.5 These two Phase 3 trials supported U.S., EU, and Japanese regulatory filings of both rozanolixizumab-noli and zilucoplan.
ZILBRYSQ was recently 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) antibody-positive,6 following FDA approval of RYSTIGGO for treatment of gMG in adult patients who are anti-AChR antibody-positive or anti-muscle-specific tyrosine kinase (MuSK) antibody-positive earlier this year.7 ZILBRYSQ and RYSTIGGO were also recently approved in Japan by the Japanese Ministry of Health, Labour and Welfare (MHLW)8. Both medicines are also under review by the European Medicines Agency (EMA) for the treatment of adults with gMG, with zilucoplan having recently received a positive CHMP opinion. Responses to these submissions are expected before the end of the year.
These data further inform UCB’s innovative approach to evolving science into meaningful solutions that help improve outcomes and help address unmet needs of people living with gMG.
“Additional results being presented at AANEM 2023 and the MGFA Scientific Sessions from the MycarinG and RAISE studies and their open-label extensions demonstrate UCB’s commitment to finding treatment options that provide sustained efficacy for patients with gMG, and reinforce the depth and strength of our expanding rare disease pipeline and portfolio,” said Donatello Crocetta, Head of Global Rare Disease & Rare Medical, UCB. “Following on from our recent approvals for RYSTIGGO and ZILBRYSQ in the U.S. and Japan for the treatment of adult patients with gMG, we’re very excited to be contributing to new treatment options to people living with this rare neuromuscular disease.”
As part of ongoing work to reveal better understanding of the patient and societal burden of gMG, UCB will also present posters on real-world data providing insights into the impact of social determinants of health on treatment of people with MG and the increase in risk of gMG exacerbation and healthcare resource utilization (HCRU) associated with higher MG-ADL scores.
Further demonstrating their commitment to finding solutions for unmet needs within the gMG community, UCB will also present study designs for Phase 2/3 studies to assess zilucoplan and rozanolixizumab-noli in pediatric patients with gMG. In total, fourteen abstracts will be presented, including three as oral presentations.
“For people living with gMG, the burden of disease is great and it’s important for us as a company to build the body of evidence around the complexities of this disease,” said Kim Moran, Head of U.S. Rare Disease, UCB. “We’re committed to creating patient value by pursuing a portfolio of differentiated solutions that are aligned to the needs and objectives of the generalized myasthenia gravis community.”
About generalized Myasthenia Gravis (gMG)
gMG is a rare autoimmune disease with a global prevalence of 100–350 cases per every 1 million people.9 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.10,11
In MG, pathogenic autoantibodies can impair synaptic transmission at the neuromuscular junction (NMJ) by targeting specific proteins on the post-synaptic membrane.12 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.10,11
About zilucoplan
Zilucoplan is a once-daily SC, self-administered peptide inhibitor of complement component 5 (C5 inhibitor). As the only once-daily generalized myasthenia gravis (gMG) target therapy for self-administration by adult patients with anti acetylcholine receptor (AChR) antibody-positive gMG, zilucoplan inhibits complement-mediated damage to the neuromuscular junction through its targeted mechanism of action.13
In October 2023, ZILBRSYQ® (zilucoplan) was approved by the U.S. FDA for the treatment of gMG in adult patients who are anti-acetylcholine receptor (AchR) antibody-positive.6 In September 2023, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion recommending granting marketing authorization for zilucoplan in the European Union (EU) as an add-on to standard therapy for the treatment of gMG in adult patients who are anti-AChR antibody-positive.14 A final decision on approval in the EU is expected before the end of the year, in line with the EMA’s standard review timeline.
Also in September 2023, the Japanese Ministry of Health, Labour and Welfare (MHLW) approved ZILBRYSQ for the treatment of gMG in adult patients (only for patients who inadequately respond to steroids or other immunosuppressants).8
Zilucoplan is currently under review by the Australian Therapeutic Goods Administration (TGA) and Health Canada for the treatment of adults with gMG. Responses from regulatory agencies to these submissions are expected during H2 2023 and H1 2024.
Orphan designation was granted by the FDA in 2019 to zilucoplan for the treatment of myasthenia gravis.
About rozanolixizumab-noli
In addition to zilucoplan, UCB’s gMG portfolio includes the FDA-approved medicine RYSTIGGO® (rozanolixizumab-noli), a subcutaneously infused monoclonal antibody targeting the neonatal Fc receptor (FcRn).1,3
In June 2023, RYSTIGGO was approved by the FDA, for the treatment of gMG in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody-positive, having been granted Priority Review for its Biologic License Application (BLA).7
In September 2023, RYSTIGGO was granted approval by the Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of generalized myasthenia gravis (gMG) in adult patients (only for patients who inadequately respond to steroids or other immunosuppressants).8
Rozanolixizumab is currently under review by the European Medicines Agency (EMA), the Center of Drug Evaluation of the China National Medical Products Administration, the Australian Therapeutic Goods Administration (TGA), Health Canada and Switzerland (Swissmedic) for the treatment of adults with gMG. Responses from regulatory agencies to these submissions are expected during H2 2023 and H1 2024.
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 8,600 people in approximately 40 countries, the company generated revenue of €5.5 billion in 2022. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news.
Contacts:
U.S. Rare Disease Communications
Daphne Teo
T +1.770.880.7655
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Global Rare Disease Communications
Jim Baxter
T +32.2.473.78.85.01
[email protected]
Corporate Communications, Media Relations
Laurent Schots
T +32.2.559.92.64
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Investor Relations
Antje Witte
T +32.2.559.94.14
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