Daily Newsletter

26 March 2024

Daily Newsletter

26 March 2024

FDA grants approval for Alexion’s NMOSD treatment

The latest approval for Ultomiris is based on positive data from the Phase III CHAMPION-NMOSD trial.

Vishnu Priyan March 26 2024

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.

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.

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.

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.

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