AstraZeneca Seeks Expanded Use For Soliris As First Targeted Therapy For Pediatric Patients With Autoimmune Neuromuscular Disease

The European Union has recommended for marketing authorization of AstraZeneca Plc's AZN Soliris (eculizumab) for expanded use in refractory generalized myasthenia gravis (gMG) in children and adolescents.

gMG is a rare, chronic autoimmune neuromuscular disease causing severe weakness and loss of muscle function.

Soliris was first approved in the EU in 2017 for certain adults with gMG and is also approved for certain adults with gMG in the U.S., China, and Japan. 

If approved, Soliris would be the first targeted therapy for pediatric patients with refractory gMG in the EU.

Positive opinion based on Phase 3 trial demonstrating clinical benefit in pediatric patients with refractory gMG.

In the trial, Soliris demonstrated clinical benefit in pediatric patients six years and older with refractory gMG who previously failed immunosuppressive treatment and continued to experience significant unresolved disease symptoms. 

Soliris showed significant improvement in the primary endpoint of change from baseline in Quantitative Myasthenia Gravis (QMG) total score at week 26, a physician-reported scale assessing disease severity and function.

Last week, the FDA approved Argenx SE's ARGX Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc), an injection for subcutaneous (SC) use for gMG in adult patients who are anti-acetylcholine receptor antibody positive. These patients represent approximately 85% of the total gMG population.

Price Action: AZN shares are up 0.57% at $72.53 during the premarket session on the last check Monday.