Agios Announces FDA Orphan Drug Designation Granted to Mitapivat for Treatment of Thalassemia
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.
- 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.
- Mitapivat was previously granted orphan drug designation by the FDA and the European Medicines Agency for pyruvate kinase (PK) deficiency, a rare, debilitating, hemolytic anemia.
- Agios is conducting a Phase 2 study evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of treatment with mitapivat in adults with non-transfusion-dependent - and -thalassemia (NTDT).
- For example, a positive opinion on Agios application for orphan drug designation for mitapivat is not a guarantee of approval.