Study: UPLINZA for Neuromyelitis Optica Spectrum Disorder

The American Academy of Neurology (AAN) 2022 Annual Meeting is held from April 2nd until the 7th in Seattle, with its virtual counterpart happening on April 24th to the 26th. At this meeting, data from the Phase 3 N-MOmentum trial was presented pertaining to UPLINZA for neuromyelitis optica spectrum disorder (NMOSD). You can read more about this data here.


Neuromyelitis optica spectrum disorder is a rare disorder characterized by inflammation of the spinal cord and optic nerve. Symptoms appear in the form of attacks, which are followed by periods of remission. Remission can range anywhere from days to years. Symptoms include:

  • Eye pain
  • Vision loss
  • Changes in sensory, motor, and autonomic functions
  • Paralysis of the lower limbs
  • Sensory loss
  • Stiffness
  • Headaches
  • Pain in the limbs and/or back
  • Changes in deep tendon reflexes
  • Diminished control of the bowels and bladder

Medical unprofessional are unsure of the exact cause of NMOSD, but they categorize it as an autoimmune disorder. This means that the immune system mistakenly attacks the body rather than foreign invaders. In terms of treatments, the FDA has approved three options for this condition: UPLINZA, Enspryng, and Soliris. In the case of an attack, doctors will treat patients with high doses of corticosteroids, plasma exchange, and immunosuppressive drugs.


Horizon Therapeutics presented the data from the N-MOmentum trial, which demonstrated UPLINZA’s efficacy and safety. This data – along with its FDA approval – is what prompts the company to describe it as

the first and only FDA-approved CD19+ B-cell-depleting monotherapy proven to reduce the risk of attacks in adults with NMOSD who are anti-aquaporin-4 (AQP4) antibody positive.

The Phase 3 trial was randomized and placebo-controlled, with participants all having recently diagnosed AQP4 antibody positive NMOSD. After their first attack, they received UPLINZA for 28 weeks and were compared to NMOSD patients who have had 2+ attacks. Important results gathered from the trial include:

  • Of the newly diagnosed NMOSD patients who had only had one prior attack being treated with UPLINZA, 4.2% experienced an attack
    • This can be compared to 23.1% of the control group
  • Of those who had experienced more than one prior attack being treated with UPLINZA, 12.4% had an attack
    • This can be compared to 48.7% in those receiving a placebo
  • Consistent safety profile
  • Between the two groups (those who had only experienced one prior attack versus those who had experienced two or more), there was no significant differences in worsening Expanded Disability Status Scale (EDSS) or attacks

Looking Forward

In the near future, Horizon also plans to share long-term extension (LTE) data focusing on pain management. It will build on the Phase 3 data already released. Because pain is a major symptom of NMOSD, a treatment that is able to reduce it will be very helpful to patients.

Overall, this data shows that UPLINZA is a safe and effective treatment for NMOSD, which fills an unmet need for this rare disease community.

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