FDA Grants Agios’ Sickle Cell Drug Orphan Status

The FDA has granted Cambridge, Mass.-based Agios Pharmaceuticals’ mitapivat an Orphan Drug designation for treating sickle cell disease.

The small molecule drug is expected to enter late-stage trials next year. The treatment previously received Orphan Drug status from the FDA for treating pyruvate kinase (PK) deficiency and for thalassemia. The European Medicines Agency (EMA) previously granted it Orphan Drug status for PK deficiency.

The company is also researching mitapivat’s ability to “improve red blood cell energy, health and longevity,” said Chris Bowden, Agios’ chief medical officer.