The biopharmaceutical company UCB has announced that the European Commission has granted marketing authorization in Europe for rozanolixizumab, as an add-on to standard therapy, for the treatment of generalized myasthenia gravis (gMG) in antibody-positive adult patients anti-acetylcholine (AChR) or anti-muscle-specific tyrosine kinase (MuSK). Rozanolixizumab 140 mg/ml, solution for injection – we read in a note – is a humanized IgG4 monoclonal antibody, which binds to the neonatal Fc receptor (FcRn), determining the reduction of circulating IgG. This is the first therapy approved in Europe for adult patients with gMG positive for both anti-AChR antibodies and anti-MuSK antibodies, the two most common subtypes of the disease.
The approval of this orphan drug is based on the results of the pivotal phase III MycarinG study in gMG, published in The Lancet Neurology in May 2023, which demonstrated that treatment with rozanolixizumab led to statistically significant and clinically relevant improvements in specific outcomes of disease compared to placebo, including an improvement in performing daily activities such as breathing, speaking, swallowing and the ability to get up from a chair. Rozanolixizumab – the note reports – is the first therapy approved in Europe for adults with gMG positive for AChR or MuSK antibodies, the two most common antibody subtypes of the disease.
European approval also for zilucoplan as an additional therapy to the standard one, for the treatment of generalized myasthenia gravis in adult patients positive for anti-acetylcholine receptor (AChR) antibodies – reports the note – Zilucoplan is a peptide inhibitor of component 5 of the complement (C5 inhibitor), to be self-administered once a day, subcutaneously for the treatment of gMG. The approval is supported by the pivotal phase III Raise study in gMG, which demonstrated that zilucoplan brought rapid, consistent and clinically relevant benefits in gMG-specific efficacy outcomes compared to placebo.
Generalized myasthenia gravis is a rare, autoimmune disease with a worldwide prevalence of 100-350 cases per million people. People affected by gMG may experience a range of symptoms, including severe muscle weakness, which can lead to double vision, droopy eyelids, difficulty swallowing, chewing and speaking, as well as life-threatening weakness of the breathing muscles.
“With the approval of rozanolixizumab by the European Commission, together with the recent approval of zilucoplan, our portfolio of medicines for gMG can now enter clinical practice across Europe. This is another important milestone in line with our mission to offer new therapeutic solutions to the community of patients living with gMG – declares Jean-Christophe Tellier, CEO of UCB – We believe that there are still many unmet needs on the part of patients, which can be answered thanks to therapeutic options differentiated, generally well tolerated and effective, addressing key aspects of the pathophysiology of gMG. We thank the patients, caregivers and investigators who participated in our clinical studies and who shared their knowledge with us. I would also like to thank our employees and collaborators who, with their dedication, energy and passion, contributed to this important result.”
Regarding the approval of zilucoplan by the European Commission, “UCB has taken another important step forward in offering value for patients with gMG, offering clinicians the opportunity to counteract complement activation in gMG, with a once-daily subcutaneous self-administration treatment option – underlines Tellier – In addition to the EC approval for our neonatal FC receptor (FcRn) blocker, rozanolixizumab, and the approvals of zilucoplan and rozanolixizumab in the United States United States and Japan for the treatment of adults with gMG, our unique and differentiated portfolio of medicines reinforces UCB's commitment to redefining treatment expectations for the generalized myasthenia gravis patient community. We are extremely grateful to the patients , to the caregivers and experimenters”, who “participated in our clinical studies, as well as to our employees and collaborators, whose dedication and passion made this important result possible” he concludes.
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