RYSTIGGO[®]▼(rozanolixizumab), for generalized myasthenia gravis (gMG), receives EU approval for two new administration methods

Brussels (Belgium) 31 January, 2025 – 07:00 AM (CET) – UCB, a global biopharmaceutical company, today announced the CHMP (Committee for Medicinal Products for Human Use) has issued a positive opinion for the self-administration of RYSTIGGO^® (rozanolixizumab) via an infusion (syringe pump) or a new manual push syringe method, after training from a healthcare professional. 

In the EU, rozanolixizumab 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) or anti-muscle-specific tyrosine kinase (MuSK) antibody-positive.¹

“For people living with gMG, unpredictable symptoms can have a significant impact on daily life, leading to patients feeling vulnerable and lacking control. Subcutaneous self-administration may help address these challenges, enhancing patient autonomy and satisfaction by reducing the need for frequent clinic visits,” said Donatello Crocetta, Chief Medical Officer and Head of Global Medical Affairs, UCB. “We welcome the EU approval for self-administration of rozanolixizumab in Europe, marking another significant step forward in our ongoing commitment to improving the lives of people living with gMG.”  

This approval is based on several studies, including a Phase 3, open-label, crossover study to evaluate the ability of patients with generalized myasthenia gravis (gMG) to successfully self-administer rozanolixizumab after training in the self-administration technique using the syringe driver and manual push methods.³ The manual push method allows administration at a flow rate that is comfortable for the patient to accommodate individual preferences.¹ In clinical trials, infusion times by manual push for rozanolixizumab ranged from 1 to 30 minutes with a median infusion time of just 5 minutes per patient. This range of infusion times may serve as a guide when training the patient or caregiver.¹

Self-administration of rozanolixizumab is also being reviewed by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA), reflecting UCB's global efforts to address the diverse, unmet needs of the gMG community. 

Currently, rozanolixizumab is administered by the patient’s clinician as a subcutaneous infusion once weekly for six weeks using infusion pump/syringe pumps, and the recommended total weekly dose of rozanolixizumab is based on the patient’s body weight.¹

About rozanolixizumab
Rozanolixizumab 140 mg/ml solution for injection* is a subcutaneously administered, humanized IgG4 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.⁴

In January 2024, the European Commission (EC) granted a marketing authorization for RYSTIGGO® (rozanolixizumab) as an add-on to standard therapy 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.¹

* Each ml of solution for injection contains 140 mg of rozanolixizumab. One vial of 2 ml contains 280 mg of rozanolixizumab. One vial of 3 ml contains 420 mg of rozanolixizumab. One vial of 4 ml contains 560 mg of rozanolixizumab. One vial of 6 ml contains 840 mg of rozanolixizumab.

This positive opinion is a type II variation to the terms of the marketing authorisation.

About generalized myasthenia gravis (gMG)

gMG is a rare autoimmune neuromuscular junction disease with a global prevalence of 100–350 cases per 1 million people.⁵ 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.^6,7

In MG, pathogenic autoantibodies can impair synaptic transmission at the neuromuscular junction (NMJ) by targeting specific proteins on the post-synaptic membrane.⁸ This disrupts the ability of the nerves to stimulate the muscle and results in a weaker contraction.⁹ gMG can occur in any race, gender or age.⁶ 

About the MG0020 study³

MG0020 was a Phase 3, open-label, randomized, two-period, two-sequence crossover study to evaluate the ability of study participants with generalized myasthenia gravis (gMG) to successfully self-administer rozanolixizumab after training in the self-administration technique using the syringe driver and manual push methods. For more information about the trial, visit https://clinicaltrials.gov/study/NCT05681715.

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 approximately 9,000 people in approximately 40 countries, the company generated revenue of € 5.3 billion in 2023. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news. 

Important Safety Information about RYSTIGGO^®▼ (rozanolixizumab) in the EU/EEA¹*
▼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. 

The most commonly reported adverse reactions were headache (48.4%), diarrhoea (25.0%) and pyrexia (12.5%). The adverse reactions from clinical studies in gMG are as follows: Very common (≥1/10) headache, diarrhoea, and pyrexia; Common (≥1/100 to <1/10) upper respiratory tract infections, including cases of nasopharyngitis, rash, angioedema, arthralgia, and injection site reactions; Not known, aseptic meningitis (from spontaneous post-marketing reporting). In MG0003, headache was the most common reaction reported in 31 (48.4%) and 13 (19.4%) of the patients treated with rozanolixizumab and placebo, respectively. All headaches, except 1 (1.6%) severe headache, were either mild (28.1% [n=18]) or moderate (18.8% [n=12]) and there was no increase in incidences of headache with repeated cyclic treatment.

Rozanolixizumab is contraindicated in patients with hypersensitivity to the active substance or to any of the excipients.

Treatment with rozanolixizumab in patients with impending or manifest myasthenic crisis has not been studied. Aseptic meningitis (drug induced aseptic meningitis) has been reported following rozanolixizumab treatment. If symptoms consistent with aseptic meningitis (headache, pyrexia, neck stiffness, nausea, vomiting) occur, diagnostic workup and treatment should be initiated as per standard of care.

As rozanolixizumab causes transient reduction in IgG levels the risk of infections may increase. Treatment with rozanolixizumab should not be initiated in patients with a clinically important active infection until the infection resolves or is adequately treated. During treatment with rozanolixizumab, clinical signs and symptoms of infections should be monitored. If a clinically important active infection occurs, withholding rozanolixizumab until the infection has resolved should be considered.

Infusion reactions such as rash or angioedema may occur. In the clinical trial, these were mild to moderate. Patients should be monitored during treatment with rozanolixizumab and for 15 minutes after the administration is complete for clinical signs and symptoms of hypersensitivity reactions. If a hypersensitivity reaction occurs during administration, rozanolixizumab infusion should be discontinued and appropriate measures should be initiated if needed. Once resolved, administration may be resumed.

Immunisation with vaccines during rozanolixizumab therapy has not been studied. The safety of immunisation with live or live-attenuated vaccines and the response to immunisation with vaccines are unknown. All vaccines should be administered according to immunisation guidelines and at least 4 weeks before initiation of treatment. For patients that are on treatment, vaccination with live or live attenuated vaccines is not recommended. For all other vaccines, they should take place at least 2 weeks after the last infusion of a treatment cycle and 4 weeks before initiating the next cycle.

This medicinal product contains 29 mg of proline in each ml. The use in patients suffering from hyperprolinaemia should be restricted to cases where no alternative treatment is available. This medicinal product contains 0.3 mg of polysorbate 80 in each ml. Polysorbates may cause allergic reactions. 

Please consult the full prescribing information in relation to other side effects, full safety and prescribing information. https://www.ema.europa.eu/ 

*EU is an abbreviation for the European Union. EEA is an abbreviation for the European Economic Area. 

Forward-looking statements

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References:

1. CHMP opinion on Rozanolixizumab EU SmPC. 16 January 2025. https://www.ema.europa.eu/en/documents/agenda/agenda-chmp-meeting-27-30-january-2025_en.pdf. Accessed January 2025. 
    
2. Menon D, Sarpong E, Bril V. Practical Aspects of Transitioning from Intravenous to Subcutaneous Immunoglobulin Therapy in Neuromuscular Disorders. Can J Neurol Sci. 2022;49(2):161–167.
3. ClinicalTrials.gov. A Phase 3, Open-label, Crossover Study to Evaluate Self-administration of Rozanolixizumab by Study Participants With Generalized Myasthenia Gravis (gMG). https://clinicaltrials.gov/study/NCT05681715. Accessed January 2025.
4. Bril V, et al. Safety and efficacy of rozanolixizumab in patients with generalized myasthenia gravis (MycarinG): a randomised, double-blind, placebo controlled, adaptive phase 3 study. Lancet Neurol. 2023;22(5):383-94.
5. Punga AR, et al. Epidemiology, diagnostics, and biomarkers of autoimmune neuromuscular junction disorders. Lancet Neurol. 2022;21(2):176-88.
6. National Institute of Neurological Disorders and Stroke. 2022. Myasthenia Gravis Fact Sheet. https://www.ninds.nih.gov/myasthenia-gravis-fact-sheet. Accessed January 2025.
7. Myasthenia Gravis Foundation of America. Overview of MG. https://myasthenia.org/understanding-mg/overview-mg. Accessed January 2025.
8. Juel VC, Massey JM. Myasthenia gravis. Orphanet J Rare Dis. 2007;2:44.
9. Howard JF Jr. Myasthenia gravis: The role of complement at the neuromuscular junction. Ann N Y Acad Sci. 2018;1412(1):113‒28.