Amgen’s Uplizna now approved in EU to treat most adults with gMG
Infusion therapy OK'd for use in AChR and MuSK positive generalized MG
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- Uplizna, an infusion therapy from Amgen, is now approved in the European Union to treat generalized myasthenia gravis.
- Already approved in the U.S., it treats adults with gMG positive for AChR or MuSK antibodies.
- Use of Uplizna is expected to reduce the need for corticosteroids among patients.
Uplizna (inebilizumab), Amgen’s infusion therapy for adults with generalized myasthenia gravis (gMG) who are positive for antibodies targeting acetylcholine receptor (AChR) or muscle-specific kinase (MuSK), is now approved for use in the European Union.
The decision by the European Commission — which applies to all EU member states and Iceland, Liechtenstein, and Norway — comes scarcely two months after Uplizna was approved in the U.S. for the same indication.
Amgen noted in a company press release that Uplizna “demonstrates durable disease control with twice-yearly dosing” and, importantly, may help cut the need for corticosteroids in people with gMG.
“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, MD, PhD, Amgen’s vice president of medical affairs.
Corticosteroids, anti-inflammatory medications often simply called steroids, are a commonly used treatment for managing gMG. However, their use, especially long term, is associated with problematic side effects.
In gMG — the most common and severe type of myasthenia gravis (MG) — immune cells called B-cells produce self-reactive antibodies that interfere with the communication between nerve and muscle cells. This results in symptoms such as fatigue and muscle weakness throughout the body. The most common MG-causing antibodies target the proteins AChR or MuSK.
Using Uplizna expected to cut need for steroids
Uplizna works by targeting CD19, a protein highly present at the surface of antibody-producing B-cells. This promotes B-cell death, thereby reducing disease-driving antibody levels. The therapy’s use is expected to ultimately ease symptoms and reduce the need for corticosteroids.
“Uplizna offers a new approach to treating gMG by selectively targeting CD19-positive B cells, which play a key role in disease [development],” said John Vissing, MD, a professor of neurology and director of the Copenhagen Neuromuscular Center, Rigshospitalet, at the University of Copenhagen in Denmark. “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.”
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 medication is also approved for other B-cell-driven diseases, such as neuromyelitis optica spectrum disorder and IgG4-related disease. It’s given intravenously, or by infusion into the bloodstream: The first two doses are given two weeks apart, and subsequent doses are given every six months.
According to Sanz Rodriguez, Uplizna may help ease the treatment burden for patients.
“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,” Sanz Rodriguez said.
In clinical trials, therapy eased impact of gMG on daily life
Uplizna’s approvals for gMG were based primarily on data from the global Phase 3 MINT clinical trial (NCT04524273). The study tested Uplizna against a placebo in 238 adults with gMG who were positive for anti-AChR or anti-MuSK antibodies.
The results showed that, after about six months, Uplizna was significantly better than the placebo at lowering scores on the MG Activities of Daily Living (MG-ADL), which measures MG’s impact in a patient’s day-to-day life and whose higher scores indicate a higher impact.
Uplizna was also superior to the placebo in reducing Quantitative MG (QMG) scores, a clinician-rated measure of muscle weakness in which higher scores indicate more severe disease.
Consistent results on both MG-ADL and QMG scores were seen in participants positive for anti-MuSK or anti-AChR after six months. Both scores continued to show greater reductions relative to the placebo after a full year of treatment among patients with anti-AChR antibodies.
In addition, 87% of Uplizna-treated participants and 85% of those on the placebo were able to lower their daily corticosteroid dose to 5 mg or less after six months.
Common side effects of Uplizna in gMG patients include headache and infusion-related reactions.
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