> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# FDA Designates Agios Mitapivat Orphan Drug
- URL: https://www.fdaweb.com/fda-designates-agios-mitapivat-orphan-drug/
- Published: 2020-11-13T12:00:00.000Z
- Updated: 2026-09-14T16:36:20.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5148086

FDA has granted orphan drug designation for Agios Pharmaceuticals’ first-in-class pyruvate kinase R (PKR) activator mitapivat for treating patients with sickle cell disease. The company [says](https://www.marketwatch.com/press-release/agios-announces-fda-orphan-drug-designation-granted-to-mitapivat-for-treatment-of-sickle-cell-disease-2020-11-12-16184314?tesla=y&ref=fdaweb.com) mitapivat is an investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes. It previously was given orphan drug designation for treating pyruvate kinase deficiency and for treating thalassemia.