Soliris OK’d for children with generalized myasthenia gravis
Therapy initially approved for adults in US in 2017
The U.S. Food and Drug Administration (FDA) has approved the expanded use of Soliris (eculizumab) for treating children with generalized myasthenia gravis (gMG), ages 6 and older, who are positive for antibodies against the acetylcholine receptor (AChR).
The infusion therapy marketed by Alexion, AstraZeneca Rare Disease was first approved in the U.S. in 2017 for adults. The label expansion now makes Soliris the only treatment approved for pediatric gMG patients in the U.S. It’s also approved in Europe and Japan for adult and pediatric patients who’ve responded inadequately to other therapies.
“This approval represents a major advancement in the treatment of pediatric myasthenia gravis and provides hope to families navigating this complex disease,” Sharon Hesterlee, PhD, chief research officer for the Muscular Dystrophy Association (MDA), said in a press release. “The availability of Soliris for children underscores the importance of continued research and innovation in neuromuscular disease treatments.”
The treatment will be given to pediatric patients via infusions into a vein in a weight-based dose. After a loading dose period, infusions are given once every two weeks. Infusions take about 35 minutes in adults, but can take one to four hours to complete in children.
Per the prescribing label, Soliris is only available through a restricted Risk Evaluation Management Strategy (REMS) program because it raises the risk of serious and life-threatening meningococcal infections caused by the Neisseria meningitidis bacteria. The program stipulates that all patients should be vaccinated against meningococcal disease at least two weeks before starting treatment, unless the risks of delaying treatment outweigh the potential risks of developing a serious infection. Patients with unresolved serious N. meningitidis infections shouldn’t use Soliris.
In gMG, the immune system generates self-reactive antibodies that mistakenly attack healthy proteins necessary for nerve-muscle communication, most often AChRs, leading to symptoms of muscle weakness and fatigue. The activation of the immune system’s complement signaling cascade is implicated in these harmful immune responses.
Soliris for generalized myasthenia gravis
Soliris is an antibody-based treatment that’s designed to inhibit complement activation by binding to a complement protein called C5. By limiting the damaging immune activity believed to contribute to gMG, the treatment seeks to ease disease symptoms.
Soliris is also used to treat other complement-mediated diseases, including paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and neuromyelitis optica spectrum disorder.
The label expansion is backed by clinical trial data in adults and safety and pharmacological data in children. An open-label Phase 3 clinical trial (NCT03759366) tested the treatment in 11 children, ages 12-17, with AChR-positive gMG who’d inadequately responded to or poorly tolerated other treatments. The children received weight-based doses of Soliris for about six months.
The results demonstrated reduced disease burden after six months, with improvements in the scores of both patient- and clinician-reported measures of disease severity. The treatment’s safety profile in children was similar to adults.
“The approval of Soliris for pediatric myasthenia gravis is a major milestone — proof of how far science has come,” said Donald S. Wood, PhD, president and CEO of the MDA. “By funding research, advocating for the needs of the community, and working alongside our industry partners, MDA and our colleagues at organizations worldwide are pushing the boundaries to bring life-changing therapies to all people living with neuromuscular diseases.”
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