Sanofi posts phase 2 win for efdoralprin alfa in AATD emphysema

Sanofi said new phase 2 data from its ElevAATe trial showed efdoralprin alfa outperformed standard plasma-derived augmentation therapy in adults with alpha-1 antitrypsin deficiency-related emphysema, with results presented May 18, 2026, at the American Thoracic Society meeting in Orlando. In the 97-patient, randomized, double-blind study, the recombinant therapy given every three weeks produced more than three times greater increases in functional alpha-1 antitrypsin trough levels than weekly plasma-derived therapy, while maintaining levels above the normal threshold for 100% of study days versus 40.8% with standard care. Sanofi said all key secondary endpoints were met, and the safety profile was comparable across arms, with no treatment-emergent adverse events leading to permanent discontinuation. The company is now discussing next steps with global regulators. (sanofi.com)

Why it matters: For veterinary professionals tracking translational respiratory and rare-disease science, this is a notable example of a recombinant replacement strategy challenging a decades-old plasma-derived standard. AATD is an underdiagnosed inherited disorder, and current human care still relies on augmentation therapy approaches introduced in the late 1980s. Broader literature describes persistent delays in diagnosis and an active search for newer therapeutic platforms, including recombinant approaches that may improve durability and dosing convenience. That kind of platform shift can shape thinking across comparative medicine, biologics development, and long-term protein replacement strategies more broadly. (sanofi.com)

What to watch: Watch for regulatory guidance, a possible US submission in the second half of 2026, and longer-term open-label data from the ElevAATe extension study. (sanofi.com)