FDA Grants Orphan Drug Designation to Mitapivat for Treatment of Thalassaemia

Agios Pharmaceuticals Inc. announced that 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 NTDT α- and β-thalassaemia.

Mitapivat is an investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes. It was previously given Orphan Drug Designation by the FDA and the European Medicines Agency (EMA) for pyruvate kinase (PK) deficiency, a rare, debilitating, hemolytic anaemia.

The FDA Orphan Drug Designation 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. Therefore, this means that the drug has not been given approval for use by any regulatory authority (read more on how the FDA Drug Approval Process works).

Agios is conducting at the moment a Phase 2 study evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of treatment with Mitapivat in adults with non-transfusion-dependent β- and α-thalassaemia (NTDT). The trial is fully enrolled, and the primary endpoint is hemoglobin response. Preliminary Phase 2 data establishing proof-of-concept for Mitapivat in thalassaemia were disclosed at the end of 2019, and updated data from this trial will be presented at the 25th European Hematology Association (EHA) Annual Congress, which is being held virtually on June 11-14, 2020.

Read the Full Agios Press Release on Mitapivat HERE