PK Deficiency Clinical Program
PK Deficiency Clinical Program
Committed to Helping Advance
the Understanding of PK Deficiency

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.

Agios‑Sponsored Studies in PK Deficiency

Mitapivat (AG-348) is an investigational compound currently being developed as part of Agios' PK deficiency clinical program.

Phase 3 Clinical Studies
Now Enrolling

Two global pivotal (phase 3) trials for mitapivat in adults with PK deficiency are actively recruiting patients:

Activate Logo

A 1:1 randomized, placebo-controlled trial of 80 patients who do not receive regular transfusions (NCT03548220).

The primary objective of the ACTIVATE Phase 3 study is to evaluate the efficacy of treatment with mitapivat, as assessed by a sustained increase in hemoglobin (Hb) concentrations.

Activate Logo T

A single-arm open-label trial of up to 40 regularly transfused patients (NCT03559699).

The primary objective of the ACTIVATE-T Phase 3 study is to evaluate the efficacy of treatment with mitapivat, as assessed by the reduction in transfusion burden.

Phase 3: Program Resources

View an Info Sheet on the ACTIVATE and ACTIVATE-T trials.


Go to the study website and find information for healthcare professionals and patients.


Registry Now Enrolling 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.

Go to Website

View an Info Card on the Peak Registry.


Learn more at and to sign up for updates from Agios.

Contact Agios Medical Affairs to learn about the Agios-sponsored studies shown here.
Phone: 833-228-8474

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.

  1. Grace RF, Zanella A, Neufeld EJ, Morton DH, Eber S, Yaish H, Glader B. Erythrocyte Pyruvate Kinase Deficiency: 2015 Status Report. Am J Hematol. 2015;90(9):825-30.

Stay Informed: Sign up for updates from Agios

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By completing and submitting this form, you agree to allow Agios and its agents to collect the information provided and to contact you regarding its PK deficiency program and related disease information. Agios will not sell or otherwise share the information you provide other than with other companies or organizations acting as our agent to administer this website or to assist in providing the information you have requested.