Research & Development
Ionis and AstraZeneca's eplontersen granted FDA Fast Track designation for transthyretin-mediated amyloid cardiomyopathy
8 February 2024 -

Biotechnology company Ionis Pharmaceuticals Inc (Nasdaq:IONS) announced on Thursday that the US Food and Drug Administration (FDA) has granted Fast Track designation to eplontersen, an investigational therapy co-developed by Ionis and pharmaceutical company AstraZeneca plc (STO:AZN) (LON:AZN) (Nasdaq:AZN) for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults.

Fast Track designation expedites the review of drugs addressing serious conditions with unmet medical needs. Results from the ongoing CARDIO-TTRansform study, the largest in this patient population, are anticipated by next year.

Previously approved in December 2023 in the US for treating hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN) as WAINUA (eplontersen), the drug is under global development and commercialisation by AstraZeneca and Ionis. WAINUA holds Orphan Drug Designation in the US and EU for treating transthyretin-mediated amyloidosis.

ATTR-CM, caused by the accumulation of misfolded TTR protein in the cardiac muscle, leads to progressive heart failure and typically results in death within three to five years from disease onset. It encompasses both genetic and wild-type forms, with an estimated 300,000 to 500,000 patients worldwide.