Agios Pyrukynd for rare blood disorder gets EMA panel backing for approval in EU

Sep. 16, 2022 7:11 AM ETAgios Pharmaceuticals, Inc. (AGIO)By: Ravikash, SA News Editor

The European Medicines Agency (<a href='' title='eMagin Corp'>EMA</a>). The stamp and an imprint


  • A committee of the European Medicines Agency (EMA) recommended the approval of Agios Pharmaceuticals' (NASDAQ:AGIO) Pyrukynd (mitapivat) to treat adult patients with pyruvate kinase (PK) deficiency.
  • PK deficiency is a rare genetic disorder characterized by the premature destruction of red bloods, which is called hemolytic anemia.
  • The positive opinion by the EMA's Committee for Medicinal Products for Human Use (CHMP) was backed by data from a phase 3 trial called ACTIVATE.
  • If approved by the European Commission (EC), Pyrukynd will be the first approved disease-modifying therapy for European patients with PK deficiency, the company said in a Sept. 16 press release.
  • Pyrukynd was approved in the U.S. in February.

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