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Sanofi Reports Phase 2 Results for Efdoralprin Alfa in Alpha-1 Antitrypsin Deficiency

Sanofi announced that its Phase 2 Elevaate study showed efdoralprin alfa normalized and maintained functional AAT levels in patients with alpha-1 antitrypsin deficiency. All key secondary endpoints were met and the treatment was well tolerated.

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1 source·May 18, 7:37 PM(10 days ago)·1m read
Sanofi Reports Phase 2 Results for Efdoralprin Alfa in Alpha-1 Antitrypsin Deficiencybenzinga.com
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Sanofi reported results from its Phase 2 Elevaate study of efdoralprin alfa for alpha-1 antitrypsin deficiency. The study found that the treatment normalized and maintained functional AAT levels. All key secondary endpoints were met.

The safety profile was comparable to plasma-derived protein.

Three-weekly dosing achieved mean functional AAT trough levels three times greater than weekly plasma-derived protein. The treatment was well tolerated in the study.

Key Facts

Phase 2 Elevaate study
showed normalization of functional AAT levels
All key secondary endpoints
were met in the study
Three-weekly dosing
achieved mean FAAT trough levels 3x greater than weekly plasma-derived protein

Potential Impact

  1. 01

    Further clinical development of efdoralprin alfa may be considered based on the Phase 2 results.

Transparency Panel

Sources cross-referenced1
Confidence score75%
Synthesized bySubstrate AI
Word count69 words
PublishedMay 18, 2026, 7:37 PM

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