First! Eculizumab Receives EU Approval for New Indication in Pediatric Myasthenia Gravis

Jul 27, 2023 17:37 CST Updated 17:37

AstraZeneca

Biopharmaceutical Manufacturer

European Union

The European Union (German: Europäische Union; French: Union européenne), abbreviated as the EU, is headquartered in Brussels, the capital of Belgium. It evolved from the European Communities and originally had six founding member states: Germany, France, Italy, the Netherlands, Belgium, and Luxembourg. The Union currently has 28 member states and 24 official languages.In December 1991, the European Council meeting in Maastricht adopted the Treaty on European Union, commonly known as the Maastricht Treaty. On November 1, 1993, the Maastricht Treaty officially entered into force, marking the formal establishment of the European Union. In 2012, the EU was awarded the Nobel Peace Prize.Donald Tusk serves as President of the European Council, and Antonio Tajani is President of the European Parliament. Jean-Claude Juncker, former Prime Minister of Luxembourg, is President of the European Commission.The EU’s treaties have been amended multiple times, and its operations are governed by the Treaty of Lisbon. Politically, all member states are democracies (according to The Economist’s 2008 Democracy Index). Economically, it constitutes the world’s second-largest economic entity (with Germany, France, and Italy being members of the G8). Militarily, the vast majority of EU member states are also members of the North Atlantic Treaty Organization (NATO).

On July 27, AstraZeneca announced that the European Union (EU) had approved the expanded use of the complement C5 inhibitor eculizumab (Soliris) for the treatment of refractory generalized myasthenia gravis (gMG) in children and adolescents aged 6 to 17 who are positive for anti-acetylcholine receptor (AChR) antibodies (Ab+). This is the first and only targeted therapy approved in the EU for the treatment of pediatric myasthenia gravis patients.

Eculizumab is the world's first approved complement inhibitor, which inhibits the uncontrolled activation of the immune system by inhibiting terminal complement. Eculizumab was initially developed by Alexion. In December 2020, AstraZeneca acquired Alexion for $39 billion, gaining rights to eculizumab. According to AstraZeneca's financial report, global sales of eculizumab reached $3.762 billion in 2022.

In 2017, eculizumab was first approved in the EU for the treatment of certain adult patients with gMG, and it was also approved in the United States, China, and Japan for the treatment of certain adult patients with gMG. Currently, marketing applications for the use of Soliris in treating pediatric patients with gMG are being or planned to be submitted to multiple regulatory authorities.

gMG is a rare, debilitating chronic autoimmune neuromuscular disorder that leads to loss of muscle function and severe weakness. The approval by the European Commission was based on the positive opinion of the Committee for Medicinal Products for Human Use (CHMP) and the results of the Phase III study of eculizumab in pediatric patients with refractory gMG.

In the study, eculizumab demonstrated clinical efficacy in refractory gMG pediatric patients who had previously failed immunosuppressive therapy and whose disease symptoms remained unresolved. At the primary endpoint of the total Quantitative Myasthenia Gravis (QMG) score at week 26, eculizumab showed significant improvement from baseline (-5.8 [95% CI -8.4, -3.13], p<0.0004).

The efficacy and safety of eculizumab in pediatric patients aged 6 years and older are consistent with the performance of eculizumab in adult refractory gMG clinical trials. The most common adverse reactions are headache and nasopharyngitis.

John F. Brandsema, M.D., of the Children's Hospital of Philadelphia, stated: "This approval marks a significant advancement in the treatment of refractory gMG pediatric patients, who previously had no targeted therapeutic options to help manage their condition. Eculizumab demonstrated clinical efficacy in a Phase III trial and showed sustained improvement in disease severity over 26 weeks, offering the potential to enhance the quality of life and redefine disease management for children and adolescents suffering from this rare neurological disorder."

Alexion CEO Marc Dunoyer said, "The impact of gMG on children can be devastating, and these families have been waiting for a solution for a long time. The approval of our first-in-class C5 inhibitor, eculizumab, in the EU for the treatment of refractory gMG in pediatric patients fully demonstrates our efforts in delivering transformative drugs to help address the unmet medical needs of the rare disease community. Eculizumab brings hope of improved outcomes for children and adolescents affected by gMG, and we are committed to increasing access to this medication for these families as soon as possible."

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