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FDA Approves AstraZeneca’s Ultomiris for Neuromyelitis Optica Spectrum Disorder


Action marks the first FDA approval of a long-acting treatment for adult patients with neuromyelitis optica spectrum disorder who are positive for the anti-aquaporin-4 antibody.

Image Credit: Adobe Stock Images/MdBabul

Image Credit: Adobe Stock Images/MdBabul

Alexion, AstraZeneca Rare Disease announced that Ultomiris (ravulizumab-cwvz) has received FDA approval to treat adults with neuromyelitis optica spectrum disorder (NMOSD) who have previously tested positive for the anti-aquaporin-4 (AQP4) antibody. The FDA based the approval on positive data from the CHAMPION-NMOSD Phase III trial, which found that Ultomiris reduced the risk of relapse in patients by 98.6% over a 73-week treatment period. As a part of the trial, Ultomiris was compared to an external placebo arm from the previous Soliris PREVENT clinical trial.1

“Alexion has been at the forefront of innovation in NMOSD, striving to offer patients a future without fear of life-altering or even fatal relapses,” said Marc Dunoyer, CEO, Alexion, in a press release. “Building on the established efficacy of C5 inhibition for people living with AQP4 Ab+ NMOSD, we are proud to deliver a transformative, long-acting treatment option that has the potential to eliminate relapses with a convenient dosing schedule every eight weeks. We are grateful to the NMOSD community for their ongoing collaboration and input, which enables us to advance science for rare diseases.”

In May 2023, Ultomiris was given approval in Japan and the European Union (EU), also based on the aforementioned data from the CHAMPION-NMOSD Phase III trial. Common adverse effects of the treatment included COVID-19, headache, back pain, arthralgia and urinary tract infection.1-3

“Just four years since delivering the first approved NMOSD treatment to the EU, we are pleased to once again advance the treatment landscape with Ultomiris, which may eliminate relapses for people impacted by NMOSD,” said Dunoyer, in a previous press release. “This approval is the culmination of extensive work and collaboration with the NMOSD community, including patients, caregivers and healthcare providers who participated in the CHAMPION-NMOSD trial, and we’re proud to continue improving access to our innovative medicines worldwide.”

According to the Cleveland Clinic, around five in every 100,000 people are diagnosed with NMOSD globally. Additionally, it is estimated to start at a median age of 39 years. However, 18% of cases reported were either in children or elderly patients. It has also been classified as a non-familial disease and is more common in female patients.4

“C5 inhibition has been proven to offer efficacy in reducing the risk of NMOSD relapses by blocking the complement system, a part of the immune system, from attacking healthy cells in the spinal cord, optic nerve and brain,” said Sean J. Pittock, MD, director, Mayo Clinic's Center for Multiple Sclerosis and Autoimmune Neurology, Mayo's Neuroimmunology Laboratory, lead primary investigator. “With today’s FDA approval, patients now have the option of a long-acting C5 inhibitor treatment that showed zero relapses in the pivotal CHAMPION-NMOSD trial, supporting the primary goal of relapse prevention in treating NMOSD.”


1. Ultomiris approved in the US for the treatment of adults with neuromyelitis optica spectrum disorder (NMOSD). AstraZeneca. March 25, 2024. Accessed March 25, 2024. https://www.astrazeneca.com/media-centre/press-releases/2024/ultomiris-approved-in-the-us-for-nmosd.html

2. Ultomiris approved in the EU for adults with neuromyelitis optica spectrum disorder (NMOSD). AstraZeneca. May 10, 2023. Accessed March 25, 2024. https://www.astrazeneca.com/media-centre/press-releases/2023/ultomiris-approved-in-the-eu-for-adults-with-neuromyelitis-optica-spectrum-disorder-nmosd.html

3. Ultomiris approved in Japan for the prevention of relapses in patients with neuromyelitis optica spectrum disorder (NMOSD). AstraZeneca. May 26, 2023. Accessed March 25, 2024. https://www.astrazeneca.com/media-centre/press-releases/2023/ultomiris-approved-in-japan-for-the-prevention-of-relapses-in-patients-with-nmosd.html

4. Neuromyelitis Optica Spectrum Disorders. Cleveland Clinic. Accessed March 25, 2024. https://my.clevelandclinic.org/departments/neurological/depts/multiple-sclerosis/ms-approaches/neuromyelitis-optica-spectrum-disorders

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