Amgen has won European Commission approval for UPLIZNA (inebilizumab) as an add-on therapy for adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) or anti-muscle specific tyrosine kinase (MuSK) antibody positive. 

 

The approval marks a major advance in targeted treatment for this rare, chronic autoimmune disease, offering twice-yearly maintenance dosing after two initial loading doses.

 

gMG affects an estimated 56,000–123,000 people across Europe and is characterized by fluctuating muscle weakness that can severely impact daily life.

 

"This approval represents an important advancement for adults with gMG in Europe, helping address debilitating symptoms and potentially reduce the long-term use of steroids where clinically appropriate," said Cesar Sanz Rodriguez, vice president of Medical Affairs at Amgen. 

 

"With convenient twice-yearly dosing and durable efficacy in people with anti‑AChR and anti‑MuSK antibody positive gMG, UPLIZNA brings a new first-in-class approach to managing this complex disease."

 

The European decision is backed by the Myasthenia Gravis Inebilizumab Trial (MINT), the largest Phase 3 study to include both AChR+ and MuSK+ patients, and the first to feature a structured steroid-tapering protocol. By Week 26, 87.4% of UPLIZNA patients and 84.6% of placebo patients reduced steroid doses to 5 mg per day or less, highlighting the therapy’s potential to lessen long-term steroid exposure.

 

"UPLIZNA offers a new approach to treating gMG by selectively targeting CD19-positive B cells, which play a key role in disease pathology," said John Vissing, professor of neurology and director of the Copenhagen Neuromuscular Center, Rigshospitalet, at the University of Copenhagen. 

 

"The approval provides both clinicians and patients a valuable new treatment option with the potential for long-term efficacy while addressing the challenges of long-term steroid exposure."

 

The MINT trial enrolled 238 adults with gMG and demonstrated statistically significant improvements across primary and key secondary endpoints.