Eplontersen granted U.S. FDA Fast Track designation for patients with transthyretin-mediated amyloid cardiomyopathy

Key Facts

Food and Drug Administration (FDA) has granted Fast Track designation to Ionis and AstraZeneca's eplontersen, an investigational therapy for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults.
"Receiving Fast Track designation from the FDA reinforces our belief that eplontersen has the potential to be a transformational treatment for patients with ATTR-CM, which remains a progressive and fatal condition for hundreds of thousands of people worldwide despite available treatment options," said Eugene Schneider, M.D., executive vice president and chief clinical development officer, Ionis.
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 investigational medicine is currently being evaluated in the Phase 3 CARDIO-TTRansform study for hereditary or wild-type transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a progressive and fatal condition that typically leads to progressive heart failure and often death within three-to-five years from disease onset.