AstraZeneca-Ionis Eplontersen granted USFDA fast track designation for transthyretin-mediated amyloid cardiomyopathy

Sarah Walters, vice president, U.S. cardiovascular, renal and metabolic diseases, AstraZeneca said, "We are committed to accelerating innovation and addressing the spectrum of unmet patient needs in amyloidosis. Together with our partner Ionis, AstraZeneca is building on our heritage in cardiovascular disease and RNA-targeted therapeutics to bring a potential best-in-class treatment for people living with ATTR-CM."

In December 2023, eplontersen was approved in the U.S. for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN, under the brand name WAINUA (eplontersen). As part of a global development and commercialization agreement, AstraZeneca and Ionis are commercializing WAINUA for the treatment of ATTRv-PN in the U.S. and are seeking regulatory approval in Europe and other parts of the world. WAINUA was granted Orphan Drug Designation in the U.S. and in the EU for the treatment of transthyretin-mediated amyloidosis (ATTR).

The global CARDIO-TTRansform Phase 3 study of eplontersen in adults with ATTR-CM is fully enrolled with more than 1,400 patients – making it the largest study in this patient population to date. The company plans to share data from the CARDIO-TTRansform study as early as 2025.