Agios to seek accelerated approval in US of oral mitapivat for SCD

Regulatory submission plans follow meeting with FDA on new trial data

Agios Pharmaceuticals is planning to file an application with the U.S. Food and Drug Administration (FDA) in the coming months that will seek accelerated approval of its oral therapy mitapivat for sickle cell disease (SCD).

This decision by Agios follows a prefiling meeting with the regulatory agency in which the developer presented new data from its global Phase 2/3 RISE UP clinical trial (NCT05031780). The data show mitapivat significantly increased levels of hemoglobin — the oxygen-carrying protein in red blood cells — and led to other clinical benefits in people with SCD.