Eculizumab (Soliris; Alexion) is an intravenous complement C5 inhibitor originally approved by FDA and EMA in adult’s patient with anti-acetylcholine receptor antibody-positive Myasthenia Gravis (MG). This approval was based on the REGAIN study (NCT01997229) and its open-label extension (NCT02301624) results.
However, in these previous trials, data about adolescent were missing. As a result, a new phase 3, open-label, multicenter study (NCT03759366) was started, recruiting 11 adolescents, aged 12 to 17 years. After 26 weeks of therapy, patients experienced least square mean changes of –5.8 for Qualitative Myasthenia Gravis score (qMG) total score and ¬–2.3 for Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score.
During the study, adverse events (AEs) were predominantly mild to moderate in severity and mainly unrelated to eculizumab. No meningococcal infections were observed.
In conclusion, this trial showed that eculizumab was safe and effective in an adolescent MG population
Brandsema JF, Ginsberg M, Hoshino H, et al. A phase 3, open-label, multicenter study to evaluate eculizumab in adolescents with refractory myasthenia gravis. Presented at: 2023 AAN Annual Meeting; April 22-27; in Boston, MA. Abstract 001926
Key Points:
- Eculizumab was safe and effective in an adolescent MG population
References:
- Brandsema JF, Ginsberg M, Hoshino H, et al. A phase 3, open-label, multicenter study to evaluate eculizumab in adolescents with refractory myasthenia gravis. Presented at: 2023 AAN Annual Meeting; April 22-27; in Boston, MA. Abstract 001926
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