Agios Announces FDA Orphan Drug Designation Granted to Mitapivat for Treatment of Thalassemia

Agios Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s first-in-class pyruvate kinase-R (PKR) activator mitapivat for the treatment of patients with thalassemia. Mitapivat is an investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes.

“Receiving orphan drug designation is an important milestone as we continue to advance mitapivat for patients with thalassemia, a serious hemolytic anemia with limited treatment options,” said Chris Bowden, M.D., chief medical officer at Agios. “We look forward to presenting updated data from our Phase 2 study of mitapivat in both alpha- and beta-thalassemia patients at the virtual European Hematology Association Annual Congress later this week.”

The FDA’s Office of Orphan Drug Products grants orphan status to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.