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London, UK — AstraZenca Ultomiris (ravulizumab-cwvz) has been approved in the United States (US) as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD).

The approval by the US Food and Drug Administration (FDA) was based on positive results from the CHAMPION-NMOSD Phase III trial, which were published in the Annals of Neurology. In the trial, Ultomiris was compared to an external placebo arm from the pivotal Soliris PREVENT clinical trial. 

Ultomiris met the primary endpoint of time to first on-trial relapse as confirmed by an independent adjudication committee. Zero relapses were observed among Ultomiris patients with a median treatment duration of 73 weeks (relapse risk reduction: 98.6%).

Neuromyelitis Optica Spectrum Disorder (NMOSD) is a rare, chronic, and potentially life-threatening autoimmune disorder primarily affecting the central nervous system. In NMOSD, the immune system mistakenly attacks healthy cells and proteins in the body, predominantly targeting the optic nerves, which transmit visual information from the eyes to the brain, and the spinal cord.

This results in inflammation and demyelination (damage to the protective covering of nerve fibers) in these areas. The hallmark symptoms include optic neuritis, leading to pain and vision loss, and myelitis, causing weakness, numbness, or paralysis in the limbs, along with bladder and bowel dysfunction. NMOSD is distinct from multiple sclerosis (MS), with which it was previously confused, and requires different treatment approaches. Diagnosis is often based on clinical presentation, MRI findings, and the presence of specific antibodies, most notably aquaporin-4. Treatment focuses on acute attacks, prevention of relapses, and symptom management.

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