Introduction: Myasthenia gravis (MG) is a neurological B-cell mediated autoimmune disorder affecting the neuromuscular junction. MG therapeutics have always relied on non-selective immunosuppression with oral steroids and non-steroidal immunosuppressants, mainly with good clinical response. However, clinical stabilization is often reached at the cost of many troublesome side effects and up to 15% of MG patients are deemed as refractory to conventional immunosuppression. This highlights the need of a more targeted and efficacious therapeutic approach. Results from the randomized-controlled period of the CHAMPION study demonstrate a good safety, tolerability, and efficacy profile of ravulizumab compared to placebo. Like eculizumab, ravulizumab is an anti-C5 monoclonal antibody, but with an enhanced pharmacokinetic profile, that allows dosing every 8 weeks. Areas covered: We provide an overview of ravulizumab biological features and results from the phase III CHAMPION MG (NCT03920293) study. Expert opinion: Data of the CHAMPION MG trial demonstrate that ravulizumab is effective and safe in the treatment of generalized MG. Having a rapid clinical effect, with long-term clinical response, ravulizumab could represent a selective immunosuppressive drug of choice in the future therapeutic algorithm of MG, where conventional immunosuppressants slowly leave room for newer drugs with a more targeted mechanism of action.
Ravulizumab for the treatment of myasthenia gravis / Vanoli, Fiammetta; Renato, Mantegazza. - In: EXPERT OPINION ON BIOLOGICAL THERAPY. - ISSN 1744-7682. - (2023).
Ravulizumab for the treatment of myasthenia gravis.
Vanoli Fiammetta;
2023
Abstract
Introduction: Myasthenia gravis (MG) is a neurological B-cell mediated autoimmune disorder affecting the neuromuscular junction. MG therapeutics have always relied on non-selective immunosuppression with oral steroids and non-steroidal immunosuppressants, mainly with good clinical response. However, clinical stabilization is often reached at the cost of many troublesome side effects and up to 15% of MG patients are deemed as refractory to conventional immunosuppression. This highlights the need of a more targeted and efficacious therapeutic approach. Results from the randomized-controlled period of the CHAMPION study demonstrate a good safety, tolerability, and efficacy profile of ravulizumab compared to placebo. Like eculizumab, ravulizumab is an anti-C5 monoclonal antibody, but with an enhanced pharmacokinetic profile, that allows dosing every 8 weeks. Areas covered: We provide an overview of ravulizumab biological features and results from the phase III CHAMPION MG (NCT03920293) study. Expert opinion: Data of the CHAMPION MG trial demonstrate that ravulizumab is effective and safe in the treatment of generalized MG. Having a rapid clinical effect, with long-term clinical response, ravulizumab could represent a selective immunosuppressive drug of choice in the future therapeutic algorithm of MG, where conventional immunosuppressants slowly leave room for newer drugs with a more targeted mechanism of action.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.