Eculizumab to treat children with generalized myasthenia gravis

The Food and Drug Administration (FDA) has approved the expansion of the indication of Soliris (eculizumab; Alexion Pharmaceuticals, Boston, MA) to now include treatment for pediatric patients aged ≥6 years with anti-acetylcholine receptor (AChR) antibody positive generalized myasthenia gravis (gMG). Soliris, a monoclonal antibody that targets the complement cascade, was previously approved for adults with AChR positive gMG in 2017 and adults with neuromyelitis optica spectrum disorder (NMOSD) in 2019, in addition to its initial approval for paroxysmal nocturnal hemoglobinuria (PNH) and a subsequent approval for atypical hemolytic uremic syndrome (aHUS).

In an open-label, multicenter, phase 3 clinical study (NCT03759366; ECU-MG-303) 11 adolescent participants aged 12 to 17 years with AChR positive gMG received weekly treatment with Soliris. The primary endpoint was change from baseline to week 26 in Quantitative Myasthenia Gravis (QMG) total score.

At week 26:QMG total score showed a least-squares mean change of -5.8 (standard error [SE], 1.2; P=.0004).
Myasthenia Gravis–Activities of Daily Living (MG-ADL) total score, a key secondary endpoint, showed a least-squares mean change of -2.3 (SE, 0.6; P=.0017).
All other secondary endpoints were met with statistical significance, demonstrating the efficacy of Soliris for improving outcomes related to symptoms, muscle strength, ability to perform daily activities, and quality of life.
Pharmacokinetic, pharmacodynamic, and safety findings were consistent with results observed in adults treated with Soliris.
3 participants experienced serious adverse events, including myasthenia gravis (MG) worsening, MG crisis, or peritonsillar abscess, and pyrexia.

Soliris’s prescribing information includes a Boxed Warning for serious meningococcal infections caused by Neisseria meningitidis.

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