Ionis Says Eplontersen Got Fast Track Status To Treat Transthyretin-mediated Amyloid Cardiomyopathy

(RTTNews) - Ionis Pharmaceuticals, Inc. (IONS) Thursday said Fast Track designation has been granted by the Food and Drug Administration (FDA) to Ionis and AstraZeneca's eplontersen, an investigational therapy for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults.

In December 2023, eplontersen was approved in the U.S. for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis( ATTRv-PN) in adults.

AstraZeneca and Ionis are commercializing eplontersen under the brand name WAINUA for the treatment of ATTRv-PN in the U.S., and are seeking regulatory approval in Europe and other parts of the world.

A Phase 3 study of eplontersen, dubbed CARDIO-TTRansform in adults with ATTR-CM, is underway with results expected in 2025.