New Data Highlights UPLIZNA Efficacy for NMOSD

How effective is UPLIZNA on treating attacks associated with neuromyelitis optica spectrum disorder (NMOSD)? According to a news release from biopharmaceutical company Horizon Therapeutics plc (“Horizon”), an analysis highlighted that UPLIZNA can actually reduce the severity of these attacks. Ultimately, this shows the therapy’s benefits for patients while also providing researchers and physicians with a better understanding of the drug’s underlying mechanisms.

What is UPLIZNA?

UPLIZNA is a biologic therapy which is approved to treat adults with NMOSD who are anti-aquaporin-4 (AQP4) antibody positive. As an estimated 80% of those with NMOSD have these antibodies, this treatment, when approved, fulfilled a huge unmet need.

The therapy works by depleting levels of CD19+ B cells. Through this, UPLIZNA may reduce inflammation and stop astrocyte damage. Astrocytes are star-shaped glial cells found throughout the central nervous system (CNS).

Within this particular study, researchers sought to determine how UPLIZNA could effectively reduce acute NMOSD attacks. Altogether, 213 patients enrolled. Of these, 161 received UPLIZNA and the remaining patients received placebos. Findings included:

  • Slightly over half of those who received the placebo had no attacks during the trial period. Alternately, 89% (143 patients) of those who received UPLIZNA had no attacks during the trial period.
  • 2/3 of the attacks that did happen during the trial were considered to be minor, with the rest being major, when treated with UPLIZNA. In those who received the placebo, 55% of the attacks were minor and 45% were major.
  • An analysis of biomarkers like sGFAP and SNfL showed that UPLINZA helped reduce attack severity.
  • While UPLIZNA is relatively safe and well-tolerated, some adverse reactions did occur. These include urinary tract infections, joint pain and stiffness, hypogammaglobulinemia, rash, fever, headache, or nausea.

About Neuromyelitis Optica Spectrum Disorder (NMOSD)

Neuromyelitis optica spectrum disorder (NMOSD) is a rare inflammatory disorder which affects the spinal cord and optic nerves, and can also affect the brain. Overall, NMOSD is considered to be an autoimmune disorder. This means that the immune system mistakenly attacks the body – in this case, the central nervous system. Because of its presentation, NMOSD may be misdiagnosed as multiple sclerosis (MS). NMOSD is characterized by optic nerve and spinal cord inflammation and occurs in “attacks,” which are followed by periods of remission. While NMOSD affects people of all backgrounds, it is slightly more prevalent in those of African or Asian descent, and much more common in females than males. Symptoms associated with NMOSD include:

  • Optic nerve and spinal cord inflammation
  • Eye pain (in one or both eyes)
  • Vision loss (in one or both eyes)
  • Limb and spine pain
  • Mild to severe lower limb paralysis
  • Headache
  • Neck stiffness
  • Loss of bowel and bladder control
  • Nausea and vomiting
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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