Illuminating the natural history of PK deficiency, as well as potential disease-specific treatment options, is central to improving care of patients with PK deficiency,1 and is a key focus at Agios.
Mitapivat (AG-348) is an investigational compound currently being developed as part of Agios' PK deficiency clinical program.
Two global pivotal (phase 3) trials for mitapivat in adults with PK deficiency are actively recruiting patients:
The Peak Registry, a global, longitudinal study of patients with PK deficiency, has been established to better understand the full spectrum of disease variability, including impact on quality of life.
The registry is committed to furthering the understanding of PK deficiency, including disease burden.
Your participation as an enrolling site will help increase knowledge and awareness of this rare genetic disorder.
View an Info Card on the Peak Registry.
Contact Agios Medical Affairs to learn about the Agios-sponsored studies shown here.
The safety and efficacy of mitapivat has not been established. There is no guarantee that it will receive health authority approval or become commercially available in any country for the use being investigated.