Novel technology clears final hurdle in rare anemia

2 December 2020

European and American regulatory submissions for Agios Pharmaceuticals’ (Nasdaq: AGIO) mitapivat are imminent, following positive results from the Phase III ACTIVATE trial.

Agios, a specialist in cellular metabolism, is developing the first-in-class small molecule for the treatment of pyruvate kinase (PK) deficiency, a rare disease for which there are no approved therapies.

Mitapivat works by activating pyruvate kinase-R (PKR) enzymes, and has been granted orphan drug designation in the USA and in Europe in this indication.

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