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AstraZeneca says Ultomiris shows 3 years relapse-free in NMOSD

ALN

AstraZeneca PLC on Friday said new long-term clinical trial data reinforce Ultomiris as a transformative treatment for neuromyelitis optica spectrum disorder, or NMOSD, a rare autoimmune disease that can cause blindness and paralysis.

The Cambridge, England-based drugmaker, through its rare disease unit Alexion, said final results from the long-term extension of the phase III Champion-NMOSD trial showed zero relapses in patients treated with Ultomiris, also known as ravulizumab, through a median follow-up of more than three years.

The data, presented at the European Committee for Treatment & Research in Multiple Sclerosis Congress in Barcelona, were selected as one of the ‘Best of ECTRIMS 2025.’

The results showed around a 99% reduction in relapse risk, with most patients remaining clinically stable or improved and many able to reduce or discontinue other immunosuppressive therapies. The safety profile was consistent with earlier findings, AstraZeneca said.

Christophe Hotermans, head of global medical affairs at Alexion, said the findings ‘reinforce the potential of Ultomiris to eliminate relapses in most patients and elevate the standard of care.’

Sean Pittock of the Mayo Clinic, lead investigator of the trial, said patients remaining relapse-free for more than three years represented a ‘significant achievement’ in NMOSD, where each relapse can cause permanent disability.

Additional data from the NMO Spotlight Registry showed real-world use of Ultomiris and predecessor therapy Soliris reduced annualised relapse rates from 0.50 to 0.02, with no patient experiencing more than one relapse.

In Japan, a separate study found patients switching from satralizumab to Ultomiris largely remained relapse-free and reduced use of other immunosuppressants.

AstraZeneca added that its ongoing Amaze observational trial will provide further insights into clinical, biological, and social factors influencing outcomes for people living with NMOSD.

Shares in AstraZeneca opened 0.6% higher at 11,320.00 pence in London on Friday.

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