Potential "first-in-class" allosteric activator received priority review qualification

Today, Agios Pharmaceuticals announced that the US FDA has accepted the new drug application (NDA) submitted by the company for mitapivat for the treatment of adult patients with pyruvate kinase (PK) deficiency

Pyruvate kinase deficiency is a rare genetic disease that manifests as chronic hemolytic anemia due to the accelerated destruction of red blood cells

Mitapivat is a potential "first-in-class" PKR allosteric activator

▲Mitapivat's potential mechanism of action (picture source: Agios official website)

This application is supported by two pivotal Phase 3 clinical trials, ACTIVATE and ACTIVATE-T

In December last year, Agios announced that mitapivat had also reached the primary endpoint in the Phase 3 clinical trial ACTIVATE in adult patients with pyruvate kinase deficiency who do not require regular blood transfusions

Reference materials:

[1] Agios Announces FDA Acceptance and Priority Review of New Drug Application for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency.

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