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Phase 2 trial shows efdoralprin alfa outperforms standard therapy in raising protective AAT levels for lung disease patients

Company Fundamentals
18 May 2026
GlobeNewsWire
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Bullish
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A phase 2 global study revealed that efdoralprin alfa, a new recombinant protein therapy, significantly outperforms the standard plasma-derived treatment in increasing and maintaining functional alpha-1 antitrypsin (fAAT) levels in patients with alpha-1 antitrypsin deficiency (AATD)-related emphysema. Efdoralprin alfa dosed every three weeks maintained normal protective protein levels 100% of the time over 32 weeks, compared to 41% with standard therapy, potentially offering less frequent dosing and better lung protection. The therapy was well tolerated with a safety profile comparable to existing treatments. Sanofi plans to engage with regulators for next steps, with efdoralprin alfa holding fast track and orphan drug designations in the US and EU.

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