The FDA has approved a new treatment for children with generalized myasthenia gravis (gMG), a disease that causes muscle weakness and fatigue. This marks the first and only approved medication for children aged six and older with gMG who have specific antibodies associated with the condition.
Generalized myasthenia gravis occurs when the immune system mistakenly attacks the connections between nerves and muscles. This leads to the accumulation of harmful antibodies, overstimulating the immune system and damaging cells, making it more difficult for muscles to function properly.
The newly approved drug, known as eculizumab and sold under the brand name Soliris, is a monoclonal antibody that targets a specific complement protein to prevent neuromuscular damage and alleviate symptoms. Initially approved in 2007, Soliris has been used to treat adults with gMG as well as other blood and immune system disorders.
This approval represents a significant advancement in the treatment of pediatric myasthenia gravis and brings new hope to affected families. The availability of Soliris for children highlights the importance of continued research and innovation in neuromuscular disease treatments.
Soliris is administered through an intravenous (IV) drip. For adults, the infusion takes approximately 35 minutes, while for children, it can take anywhere from one to four hours. Due to the increased risk of meningococcal infections associated with this medication, it is available only through a restricted safety program known as the Risk Evaluation and Mitigation Strategy (REMS). Patients must receive a meningococcal vaccine at least two weeks before starting treatment to reduce the risk of serious infections.
The FDA’s decision to approve Soliris for pediatric use was based on clinical trial data from adult patients with gMG, as well as safety studies conducted in children. In a 26-week study involving children aged 12 to 17, the observed side effects were similar to those seen in adults. The most commonly reported side effects included bone and muscle pain.
This milestone in pediatric gMG treatment underscores the growing focus on developing targeted therapies for neuromuscular diseases. As new treatments emerge, there is greater potential to improve the quality of life for children living with this challenging condition. Families navigating pediatric myasthenia gravis now have access to an option that could provide meaningful relief and better disease management.
