The U.S. Food and Drug Administration (FDA) has approved Soliris as the first treatment for children aged six and older with generalized myasthenia gravis (gMG), a rare neuromuscular disorder. Approved on March 11, 2025, Soliris is a monoclonal antibody designed to block a specific complement protein, thereby preventing neuromuscular damage and alleviating symptoms associated with the disease. This development marks a significant advancement in the treatment of pediatric myasthenia gravis.
Soliris is administered through an intravenous (IV) drip, with the duration varying by age group. For adults, the infusion takes approximately 35 minutes, while for children, it ranges from one to four hours. The drug is available exclusively through a Risk Evaluation and Mitigation Strategy (REMS) program due to its potential to increase the risk of meningococcal infections.
Patients prescribed Soliris are required to receive a meningococcal vaccine at least two weeks prior to starting their treatment regimen. This precaution is essential to mitigate the heightened risk of infection posed by the medication. Soliris is noted for being the only approved medicine specifically targeting children with gMG who possess certain antibodies linked to the condition.
"This approval represents a major advancement in the treatment of pediatric myasthenia gravis and provides hope to families navigating this complex disease,” said Sharon Hesterlee, PhD, chief research officer at the Muscular Dystrophy Association.
The approval of Soliris for pediatric use underscores a critical step forward in addressing the needs of young patients suffering from gMG. By blocking complement proteins that contribute to neuromuscular damage, Soliris offers a means to manage symptoms effectively, providing new hope for improved quality of life.
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