Alpha-1 antitrypsin deficiency (AATD) is a genetic condition that is an underlying cause of some forms of COPD, including emphysema. A new treatment for this condition may be on the horizon.
Data from the ElevAATe phase 2 study demonstrated the superiority of efdoralprin alpha over standard-of-care therapy in achieving and maintaining normalized functional alpha-1 antitrypsin (fAAT) levels in adults with AATD-related emphysema. The results were presented at the 2026 American Thoracic Society International Conference in Orlando, Florida.
Efdoralprin alfa, dosed every three weeks, achieved mean increases in fAAT trough levels more than three times greater than plasma-derived protein (pdAAT) dosed weekly, meeting the primary endpoint of the trial, according to a press release from Sanofi, the drug’s manufacturer.
The ElevAATe phase 2 study was a double-blind, randomized trial including 97 patients with emphysema. In patients dosed every three weeks, fAAT levels remained above the normal threshold for 100% of the days during the 32-week study. That compared to 41% of days in patients on a standard-of-care augmentation therapy.
Sanofi
AATD is a rare, undiagnosed genetic condition that can cause considerable respiratory illness and is characterized by low levels of AAT, a protein that protects the lungs from damaging inflammation. Without adequate fAAT levels, patients can experience progressive deterioration of the lung tissue and may develop emphysema.
“AATD presents a persistent clinical challenge,” said Igor Barjaktarevic, MD, PhD, associate professor at the David Geffen School of Medicine at UCLA and principal investigator of the ElevAATe phase 2 study. “Widespread lack of awareness of the condition as a genetic cause of some forms of COPD leaves many patients underserved. Without treatment to address the underlying AAT protein deficiency, these patients are unable to maintain protective protein levels and become vulnerable to progressive lung disease.”
