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Sanofi’s Efdoralprin Alfa Beats Standard Therapy in Phase 2 AATD Trial

The recombinant design signals a possible plasma-free option that could ease weekly treatment burdens.

The logo of French drugmaker Sanofi is seen in Paris, France, January 9, 2026. REUTERS/Gonzalo Fuentes/File Photo

Overview

  • Sanofi reported at the American Thoracic Society meeting in Orlando that efdoralprin alfa raised trough alpha‑1 antitrypsin by 24.1 micromolar at week 32 versus 7.6 micromolar with weekly plasma therapy.
  • The 97-person ElevAATe study randomly assigned patients to efdoralprin alfa every three or four weeks or to the standard weekly plasma-derived infusion.
  • Patients on the study drug kept protective AAT protein within the normal range throughout the trial, while those on standard care did so less than half the time.
  • The therapy is made with recombinant technology, which could reduce reliance on donated plasma that limits supply for current treatments.
  • The program remains experimental and unapproved after Phase 2, following Sanofi’s 2024 $2.2 billion purchase of the asset through its Inhibrx deal.