AstraZeneca’s Alexion unit set to showcase broad rare disease data at AAN 2026

Alexion, which is AstraZeneca’s rare disease division, is set to make a significant presence at the American Academy of Neurology Annual Meeting in Chicago this April (18–22), bringing its largest neurology data package to date across three rare conditions: generalised myasthenia gravis (gMG), neurofibromatosis type 1 with plexiform neurofibromas, and neuromyelitis optica spectrum disorder.

Note that a total of 20 presentations are planned, five of which will be delivered orally.

Among the highlights is new Phase III data from the PREVAIL trial, assessing gefurulimab — an investigational self-administered weekly injection for gMG. Results show the treatment produced statistically significant improvements in disease composite scores at 26 weeks compared to placebo. If approved, gefurulimab could offer patients a more convenient, at-home treatment option for this debilitating neuromuscular condition.

On the NF1 front, two oral presentations will spotlight Koselugo. One draws on a qualitative sub-study from the KOMET trial — the only placebo-controlled Phase III study in adults with NF1-PN — showing patients reported meaningful reductions in pain, fatigue and tumour-related symptoms. A separate analysis of US insurance claims data found that Koselugo users had sustained reductions in prescription painkiller use over a three-year period.

In addition, for NMOSD the presentations will include transcriptomics data from the CHAMPION-NMOSD trial alongside real-world evidence on the long-term use of Ultomiris.

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AAN, AAN 2026, Alexion, AstraZeneca, AstraZeneca's Alexion