CHMP favors Soliris be approved in Europe to treat children with gMG

Decision awaited for refractory patients, ages 6 to 17, with anti-AChR antibodies

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A branch of the European Medicines Agency (EMA) recommended that the approval of Soliris (eculizumab) be expanded to include children and adolescents, ages 6 to 17, with generalized myasthenia gravis (gMG) who are positive for antibodies targeting the acetylcholine receptor (AChR) — the most common type of MG-driving antibody.

The recommendation, from the EMA’s Committee for Medicinal Products for Human Use (CHMP), will now be reviewed by the European Commission, which makes final decisions on therapy approvals in the European Union. The commission is not obligated to abide by a CHMP opinion, but it usually does.

“If approved, Soliris would be the first targeted treatment for paediatric patients living with refractory gMG in Europe, offering the possibility of improved outcomes and quality of life,” Marc Dunoyer, CEO of Alexion, a subsidiary of AstraZeneca and Soliris’ initial developer, said in a company press release.

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Phase 3 trial in pediatric patients supported CHMP’s favorable opinion

Soliris has been approved in the EU since 2017 as a treatment for adults with gMG who have refractory disease, meaning they have failed to respond to other treatments, and who are positive for anti-AChR antibodies. The therapy has received similar approvals in the U.S., Japan, and, most recently, in China.

Soliris is administered via an infusion directly into the bloodstream, and after an initial loading dose, infusions are given every other week. The therapy is designed to block the activation of the complement cascade, part of the immune system that is thought to play a key role in the autoimmune attack that drives MG. It specifically targets a complement protein called C5.

CHMP’s recommendation was supported by data from an open-label Phase 3 study (NCT03759366), which tested the therapy in children ages 6 to 17 with refractory, anti-AChR-positive gMG. The trial’s main goal was to evaluate how the treatment affected the scores on the Quantitative MG (QMG) scale, a physician-rated measure of MG severity, after 26 weeks (about six months).

Results showed that the average QMG score decreased significantly, by 5.8 points, suggesting that the treatment eased disease symptoms. Overall, the efficacy and safety profile of Soliris in these young patients was similar to what was seen in prior studies of adults. Most reported side effects were mild or moderate in severity, with the most common including headache and the common cold.

“Onset of gMG in paediatric patients is extremely rare and complex to manage. As the disease progresses, patients may become nonresponsive to standard treatments, leaving them at risk for serious complications,” said John Brandsema, MD, a primary study investigator at the Children’s Hospital of Philadelphia.

“The Phase III clinical trial results in patients aged 12 to 17 years with refractory gMG show the efficacy of C5 inhibition in substantially reducing disease severity and symptoms, potentially transforming how this debilitating disease is managed in certain paediatric patients,” Brandsema said.

AstraZeneca said it is preparing applications that seek to extend Soliris’ use to children with gMG in other regions.