FDA approves Soliris for patients aged 6 years and up with generalized myasthenia gravis
Key takeaways:
- The expanded application makes this the first available treatment for myasthenia gravis in children.
- Soliris is available only through a Risk Evaluation Management Program.
The FDA has approved an expanded indication of Soliris, an IV-administered monoclonal antibody for patients aged 6 years and older with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive.
According to a release from the Muscular Dystrophy Association, Soliris (eculizumab, Alexion/AstraZeneca) is the first and only treatment available to address the neuromuscular disease in this younger patient population.
Previously approved for adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor positive(AChR+), the approval was supported by clinical trial data from adults with the specific condition, as well as safety and pharmacokinetic data from a 26-week, single-arm study of 11 patients aged 12 to 17 years that showed similar adverse reactions in adolescents as adults.
“Myasthenia gravis (MG) can impact anyone at any age and is extremely devastating for young patients and their families,” Samantha Masterson, president and CEO of the Myasthenia Gravis Foundation of America, told Healio. “Through groundbreaking research and a commitment to advancing therapeutics, pediatric patients now have a new treatment option that could help them live an improved quality of life with MG.”
Administered by infusion, Soliris is solely available through a restricted Risk Evaluation Management Strategy (REMS) program. The REMS requires patients to be vaccinated against meningococcal disease at least 2 weeks ahead of treatment initiation.
Soliris was initially approved in 2007 for paroxysmal nocturnal hemoglobinuria, then subsequently approved for indications such as atypical hemolytic uremic syndrome, adult gMG with AChR antibodies and neuromyelitis optica spectrum disorder.
“This approval represents a major advancement in the treatment of pediatric myasthenia gravis and provides hope to families navigating this complex disease,” MDA chief research officer Sharon Hesterlee, PhD, said in the release. “The availability of Soliris for children underscores the importance of continued research and innovation in neuromuscular disease treatments.”