ALXN2220 Receives FDA Fast Track Designation for Transthyretin Amyloid Cardiomyopathy

Recently,AstraZeneca announced that its rare disease subsidiaryAlexionOfALXN2220 hasObtainFDA Fast TrackRecognition, used to treat transthyretin amyloid cardiomyopathy（ATTR-CM）。

ALXN2220 is a novel investigational humanized depleting monoclonal antibody designed to clear amyloid deposits. The Fast Track designation was based on the results of a Phase Ib trial published in the *New England Journal of Medicine* in 2023. In 40 patients with wild-type or variant ATTR-CM and chronic heart failure, intravenous administration of ALXN2220 improved cardiac tracer uptake and extracellular volume (a surrogate marker of cardiac amyloid load) without significant drug-related adverse events within 12 months.

Currently, Alexion has initiated an international multicenter (including China) Phase III clinical trial (DepleTTR-CM study) of ALXN2220 injection to evaluate its efficacy as an add-on therapy to standard treatment in patients with ATTR-CM. The primary endpoint is a composite endpoint consisting of all-cause mortality and the cumulative incidence of cardiovascular clinical events.

Gianluca Pirozzi, Senior Vice President and Head of Development, Regulatory, and Safety at Alexion, stated in the press release that ALXN2220, with its novel depletion mechanism capable of removing existing amyloid deposits and restoring organ function, has the potential to alter the course of ATTR-CM.