Sanofi’s Efdoralprin Alfa Gains EMA Orphan Status for Rare Lung Disease

Sanofi (EURONEXT: SAN) announced Wednesday that its investigational therapy, efdoralprin alfa, has received orphan drug designation from the European Medicines Agency (EMA) for the treatment of alpha-1 antitrypsin deficiency (AATD)–related emphysema. The designation highlights the potential of the therapy to address a rare and serious lung condition that affects a limited patient population across the European Union.

Under EU regulations, orphan designation is granted to medicines intended for diseases affecting no more than five in 10,000 people. Alpha-1 antitrypsin deficiency is a rare inherited disorder caused by low levels of the alpha-1 antitrypsin protein, which plays a critical role in protecting lung tissue from inflammation and damage. When this protective protein is insufficient, patients are at high risk of developing progressive lung disease, including chronic obstructive pulmonary disease (COPD) and emphysema.

Efdoralprin alfa is a recombinant human alpha-1 antitrypsin-Fc fusion protein designed to restore functional alpha-1 antitrypsin levels to the normal range. By inhibiting neutrophil elastase, an enzyme known to damage lung tissue, the investigational therapy aims to slow or prevent lung deterioration in patients with AATD. In the global phase 2 ElevAATe clinical trial, efdoralprin alfa demonstrated superiority over standard plasma-derived alpha-1 antitrypsin therapy when administered every three or four weeks. The study met all primary and key secondary endpoints, supporting its potential as a next-generation treatment option.

The EMA decision follows earlier regulatory support in the United States, where the U.S. Food and Drug Administration granted both fast track and orphan drug designation to efdoralprin alfa. Sanofi plans to present the full clinical data at an upcoming medical conference and will engage with global regulatory authorities to determine the next steps in development.

Approximately 235,000 people worldwide are estimated to have alpha-1 antitrypsin deficiency, yet nearly 90% remain undiagnosed. With no new therapies introduced since plasma-derived treatments were first approved in 1987, the orphan designation underscores the significant unmet medical need and the importance of innovation in rare respiratory diseases.