The efficacy and safety of the therapy was assessed in 58 adult NMOSD patients. Credit: Davizro Photography / Shutterstock.com.

The US Food and Drug Administration (FDA) has approved Alexion, AstraZeneca Rare Disease’s Ultomiris (ravulizumab-cwvz) to treat adults with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD).

This is the first and only long-acting C5 [human complement component 5 protein] inhibitor to receive such approval in the US.

Go deeper with GlobalData

Premium Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

The latest development is based on the positive outcomes from the global, open-label, multicentre CHAMPION-NMOSD Phase III trial.

It assessed the efficacy and safety of Ultomiris in 58 adult NMOSD patients from Europe, North America, Asia-Pacific and Japan.

Ultomiris was benchmarked against an external placebo group from the Soliris PREVENT clinical trial.

See Also:

The primary endpoint of time to first on-trial relapse was met, with zero relapses observed among patients receiving Ultomiris over a median treatment duration of 73 weeks.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

Ultomiris’ safety and tolerability profile aligned with earlier clinical studies and no new safety concerns were identified.

Covid-19, back pain, headache, arthralgia and urinary tract infections were the most frequently reported adverse events.

Ultomiris is already approved for specific adult patients with NMOSD in the European Union and Japan, with regulatory reviews underway in other countries.

Alexion CEO Marc Dunoyer stated: “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.

“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.”

AstraZeneca recently entered an agreement to acquire Fusion Pharmaceuticals for up to $2.4bn in a strategic move to expand its portfolio.