The Food and Drug Administration (FDA) has accepted for Priority Review the supplemental New Drug Application (sNDA) for mitapivat in sickle cell disease (SCD).
Mitapivat is an oral pyruvate kinase (PK) activator currently approved under the brand name Pyrukynd® for the treatment of hemolytic anemia in adults with PK deficiency and under AqvesmeTM for the treatment of anemia in adults with alpha- or beta-thalassemia. In the treatment of SCD, mitapivat is expected to improve red blood cell health and prevent sickling by increasing adenosine triphosphate levels and lowering typically elevated 2,3-diphosphoglycerate levels.
The sNDA is supported by data from the double-blind, placebo-controlled, phase 2/3 RISE UP study (ClinicalTrials.gov Identifier: NCT05031780), which enrolled 207 patients aged 16 years and older with SCD. To meet inclusion criteria, participants were required to have experienced 2 to 10 sickle cell pain crises (SCPCs) in the 12 months prior to screening and maintain a hemoglobin level between 5.5g/dL and 10.5g/dL during screening.