AstraZeneca’s Self-Administered Myasthenia Gravis Drug Gefurulimab Clears Phase 3 Trial

AstraZeneca announced that its investigational myasthenia gravis drug, gefurulimab, successfully met all primary and secondary endpoints in the Phase 3 PREVAIL trial135.

Gefurulimab is a complement C5 inhibiting nanobody, designed for once-weekly self-administration13.

The drug demonstrated statistically significant and clinically meaningful improvement on the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale when compared to placebo, though no specific efficacy data was released yet135.

The study involved 260 adults with anti-acetylcholine receptor antibody-positive (AChR Ab+) generalized myasthenia gravis, the most common form of the disease13.

Gefurulimab was well-tolerated, with a safety profile consistent with previous studies and no new safety signals reported35.

The drug was acquired as part of AstraZeneca’s $40 billion purchase of Alexion Pharmaceuticals and is seen as central to AstraZeneca's strategy for rare diseases3.

AstraZeneca plans to present detailed trial data at a future scientific meeting and will share results with global regulators soon13.

A once-weekly, self-administered drug offers patients greater convenience and independence compared to existing treatments3.

Sources:

1. https://www.biospace.com/drug-development/astrazenecas-claims-piii-win-with-nanobody-treatment-for-myasthenia-gravis

3. https://www.biopharmadive.com/news/astrazeneca-gefurulimab-myasthenia-gravis-study-results/753909/

5. https://seekingalpha.com/news/4471407-astra-posts-late-stage-trial-win-myasthenia-gravis-drug