• 13 Dec 2025
• Admin
• News Article

Amgen Secures FDA Approval for Uplizna in Generalized Myasthenia Gravis

Amgen has received US FDA approval for Uplizna (inebilizumab-cdon) to treat adults with generalized myasthenia gravis (gMG) who are AChR- or MuSK-antibody positive.

This approval introduces a new targeted treatment option that offers durable disease control with just two maintenance doses per year after initial loading doses.

Why This Approval Matters?

Generalized myasthenia gravis is a rare, chronic autoimmune disease that disrupts neuromuscular communication.

Patients often experience:

• Muscle weakness
• Difficulty breathing
• Problems with speech, vision, and swallowing
• Unpredictable disease flares

In the US alone, gMG affects 80,000–100,000 people, with limited long-term treatment options.

How Uplizna Works?

Uplizna is a humanized monoclonal antibody that targets CD19-positive B cells, which produce harmful autoantibodies in gMG.

By depleting these B cells, Uplizna addresses a root cause of disease, rather than only managing symptoms.

Key advantages include:

• Targeted immune modulation
• Sustained efficacy
• Twice-yearly dosing after initial treatment

Strong Phase 3 Trial Results

The FDA approval is based on results from the MINT Phase 3 trial, the largest biologic study conducted in gMG.

Key efficacy outcomes at Week 26:

• 1.9-point improvement in MG-ADL score versus placebo (p<0.0001)
• Significant improvements in muscle strength (QMG score)
• Benefits observed in both AChR+ and MuSK+ patients

Longer-term results:

• Continued improvement through 52 weeks in AChR+ patients
• Reduced disease burden with sustained symptom control

Steroid Reduction Built Into the Study

MINT was the first gMG trial to require steroid tapering.

By Week 26:

• 87.4% of Uplizna patients reduced prednisone to ≤5 mg/day
• This helps lower long-term steroid-related complications

Safety Profile

Uplizna was generally well tolerated. The most common side effects were:

• Headache
• Infusion-related reactions

No new safety concerns were identified during the trial.

A Growing Franchise for Amgen

This marks the third FDA-approved indication for Uplizna.

Previous approvals include:

• Neuromyelitis optica spectrum disorder (NMOSD)
• IgG4-related disease (IgG4-RD)

The gMG approval further strengthens Amgen’s presence in rare autoimmune diseases.

What This Means for Patients?

With Uplizna, patients gain:

• Long-lasting symptom control
• Fewer infusions per year
• Reduced reliance on steroids
• Improved daily function

For people living with gMG, this approval represents six months of treatment-free time between maintenance doses.

Bottom Line

The FDA approval of Uplizna introduces a first-in-class, B-cell–targeted therapy for generalized myasthenia gravis.

It expands treatment options, simplifies dosing, and addresses the disease at a biological level—offering meaningful progress for patients and clinicians alike.