By Counterforce Health Team —

Uplizna: A Therapy for NMOSD

Neuromyelitis optica spectrum disorder (NMOSD) is a rare, chronic autoimmune disease that affects the central nervous system — particularly the optic nerves and spinal cord. It often causes severe attacks of vision loss, muscle weakness, paralysis, and other disabling symptoms.

For many years, NMOSD was difficult to manage, and patients relied on steroids or other immunosuppressive drugs that were only partly effective.

In 2020, the U.S. Food and Drug Administration (FDA) approved Uplizna® (inebilizumab-cdon), a targeted biologic therapy that helps prevent NMOSD relapses by attacking a key part of the immune system responsible for the disease.

This approval marked a major step forward in providing long-term control and hope for people living with NMOSD.

What Is Uplizna?

Uplizna (inebilizumab-cdon) is a prescription monoclonal antibody medication used to treat:

Adults with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive.

Being “AQP4 antibody positive” means that a patient’s immune system produces antibodies that mistakenly attack a protein called aquaporin-4, found on nerve cells in the brain, spinal cord, and optic nerves. This immune attack causes inflammation and damage, leading to NMOSD symptoms.

Uplizna is designed to target and eliminate specific immune cells that drive this attack, helping reduce relapses and prevent further nerve damage.

Understanding NMOSD

NMOSD is an autoimmune demyelinating disorder, meaning the body’s immune system mistakenly attacks myelin — the protective sheath surrounding nerve fibers.

Common symptoms include:

  • Sudden vision loss in one or both eyes
  • Pain behind the eyes
  • Weakness or paralysis in the arms or legs
  • Loss of bladder or bowel control
  • Severe spasms or stiffness
  • Nausea and vomiting (when the brainstem is affected)

Each NMOSD attack can cause permanent damage, making relapse prevention essential to preserving vision and mobility.

How Uplizna Works

Uplizna targets a specific type of B cell, an immune cell that contributes to NMOSD by producing harmful AQP4 antibodies.

Here’s how it works step by step:

  1. Uplizna is a monoclonal antibody that binds to a protein called CD19, which is found on the surface of most B cells.
  2. Once bound, Uplizna marks these B cells for destruction, effectively lowering their numbers.
  3. With fewer B cells available, the body produces fewer AQP4 antibodies, which in turn reduces inflammation and nerve damage.

By controlling B cell activity, Uplizna helps prevent future attacks and slows the progression of disability.

How Uplizna Is Administered

  • Route: Intravenous (IV) infusion
  • Initial dosing: Two infusions, two weeks apart
  • Maintenance dosing: One infusion every six months thereafter

Each infusion is given in a healthcare setting and usually lasts about 90 minutes, followed by an observation period.

This twice-yearly dosing schedule makes Uplizna one of the most convenient long-term treatments available for NMOSD.

Clinical Evidence and FDA Approval

Uplizna was approved by the FDA in June 2020, based on the results of the N-MOmentum Phase 2/3 trial.

Key findings from the study:

  • Among AQP4 antibody-positive patients, Uplizna reduced the risk of relapse by 77% compared to placebo.
  • The majority of patients remained relapse-free through 28 weeks of the study period.
  • MRI scans showed less inflammation and fewer new lesions in patients treated with Uplizna.
  • Disability scores were stable or improved in most patients.

These results confirmed Uplizna’s strong ability to prevent attacks and protect the nervous system from further injury.

Benefits of Uplizna

Uplizna offers several important benefits for patients with AQP4-positive NMOSD:

  • Significantly reduces relapse risk
  • Helps preserve vision and mobility
  • Twice-yearly dosing for easier adherence
  • Targets the root cause of the disease (B cell activity)
  • Well-tolerated with manageable side effects

For many patients, Uplizna means fewer hospitalizations, less disability, and a more stable long-term outlook.

Possible Side Effects

While generally well tolerated, Uplizna can cause side effects.

Common side effects include:

  • Infusion-related reactions (fever, headache, rash, nausea during infusion)
  • Urinary tract infections (UTIs)
  • Joint pain
  • Fatigue

More serious but less common risks:

  • Infections: Because Uplizna reduces certain immune cells, there is a slightly higher risk of infections.
  • Low immunoglobulin levels: Long-term treatment may lower antibody levels in the blood; doctors may monitor these periodically.
  • Reactivation of hepatitis B virus (HBV): Screening for HBV is recommended before starting therapy.

Patients are usually pretreated with medications (like steroids or antihistamines) before infusions to minimize reactions.

Who Can Receive Uplizna

Uplizna is approved for adults with NMOSD who test positive for AQP4 antibodies.

It is not currently approved for AQP4-negative NMOSD or for other autoimmune neurological disorders such as multiple sclerosis.

Before starting treatment, patients should undergo:

  • Blood tests for AQP4 antibodies and hepatitis B
  • Vaccination review (live vaccines should be given at least 4 weeks prior to starting Uplizna)
  • Baseline blood counts and immunoglobulin levels

Monitoring and Follow-Up

During and after treatment, healthcare providers typically monitor:

  • Signs of infection
  • Immunoglobulin (IgG) levels
  • B cell counts (to confirm suppression)
  • NMOSD activity (through clinical exams and MRI if needed)

Ongoing follow-up ensures that treatment remains effective and safe over time.

Practical Considerations

  • Storage: Uplizna is stored and prepared by healthcare professionals.
  • Infusion setting: Treatments take place in an infusion clinic or hospital.
  • Lifestyle: Patients should avoid live vaccines during treatment and practice good infection prevention.
  • Support: The manufacturer offers patient assistance and reimbursement programs for eligible individuals.

The Bottom Line

Uplizna (inebilizumab-cdon) represents a major advancement in the treatment of neuromyelitis optica spectrum disorder (NMOSD). By targeting and depleting B cells that produce disease-causing antibodies, it helps prevent relapses, protect vision, and preserve neurological function.

With convenient twice-yearly dosing and strong clinical results, Uplizna has quickly become a cornerstone therapy for people living with this challenging autoimmune condition.

For patients diagnosed with AQP4-positive NMOSD, Uplizna offers not just treatment — but a path toward stability, independence, and a better quality of life.