The first-in-class C5 complement inhibitor, Soliris has been indicated for anti-acetylcholine receptor antibody-positive children and adolescents aged six to 17 years.
It is claimed to be the first and only targeted therapy to receive approval to treat patients of this age group with refractory gMG, in the European Union (EU).
In 2017, Soliris initially received approval in the EU to treat certain adults with gMG.
The latest EC’s approval follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP).
AstraZeneca’s subsidiary Alexion CEO Marc Dunoyer said: “The impact of gMG on children can be devastating, and families have long been awaiting solutions.
“This approval of our first-in-class C5 inhibitor Soliris for paediatric patients with refractory gMG in the EU exemplifies our efforts to deliver transformative medicines that help address unmet medical needs for rare disease communities.
“Soliris offers hope for improved outcomes for children and adolescents impacted by gMG and we are committed to increasing access for these families as quickly as possible.”
The regulatory approval is based on data obtained from the Phase III clinical trial of Soliris conducted in refractory gMG paediatric patients.
The 26-week multicentre, open-label trial was designed for evaluating Soliris’s efficacy and safety in 11 patients aged 12 to 17 years.
According to the findings, Soliris showed clinical benefit in refractory gMG paediatric patients who were unresponsive to immunosuppressive therapy and had significant unresolved symptoms.
It also demonstrated sustained improvements in disease severity and function over 26 weeks.