Alaa Hamed, MD, Global Head of Medical Affairs Rare Diseases at Sanofi, discusses results from the ElevAATe clinical trial of efdoralprin alfa for the treatment of patients with alpha-1 antitrypsin deficiency (AATD).
AATD is an inherited disease that causes an increased risk of having chronic obstructive pulmonary disease (COPD), liver disease, skin problems, and vasculitis. Pulmonary problems almost always occur in adults, whereas liver and skin problems may occur in adults and children. Symptoms may include shortness of breath and wheezing, repeated infections of the lungs and liver, yellow skin, fatigue, rapid heartbeat when standing, vision problems, and weight loss. However, some people with AATD do not have any problems.
AATD is caused by genetic changes in the SERPINA1 gene. The genetic changes cause too little or no working alpha-1 antitrypsin protein (AAT) to be made. AAT is made in the liver cells and sent through the bloodstream to the lungs where it helps protect the lungs from damage. Having low levels of AAT (or no AAT) may lead the lungs to become damaged. A build-up of abnormal AAT may cause liver damage.
Efdoralprin alfa is a recombinant human AAT-Fc fusion protein being investigated for treatment of adults with AATD emphysema. The treatment is designed to restore functional AAT levels to the normal range and inhibit neutrophil elastase. The ElevAATe clinical trial is a phase 2 double-blind, randomized study evaluating efdoralprin alfa versus standard-of-care plasma-derived augmentation therapy in patients with AATD emphysema. The trial has enrolled 97 patients who receive the treatment every three weeks, every four weeks, or standard-of-care once weekly.
Results were recently announced showing that efdoralprin alfa demonstrated a statistically significant greater mean increase in functional AAT levels within normal range as measured by trough concentrations at steady state compared to patients on standard-of-care at week 32. Key secondary endpoints were also met, demonstrating superior mean increase in fAAT average concentration as well as higher percentage of days above the lower limit of normal range for both 3-week and 4-week dosing groups.
Additionally, efdoralrpin alfa was well-tolerated and had a similar adverse event profile to standard-of-care. Additionally safety follow-up will be assessed in the ElevAATe open-label extensions study.
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To learn more about AATD and other rare lung conditions, visit https://checkrare.com/diseases/lung-diseases/