Agios Gets CHMP Backing of Pyrukynd in Thalassemia

Dow Jones
Oct 17
 

By Colin Kellaher

 

Agios Pharmaceuticals said a key European regulatory committee has recommended expanded approval of its Pyrukynd anemia drug for certain people with the inherited blood disorder thalassemia.

Agios on Friday said the recommendation by the European Medicines Agency's Committee for Medicinal Products for Human Use covers Pyrukynd in adults for the treatment of anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia.

The European Commission, which generally follows CHMP's advice, will now review the recommendation, with a decision expected by early next year, Agios said.

The U.S. Food and Drug Administration is currently reviewing Pyrukynd in thalassemia, with a target action date of Dec. 7

The drug is already approved in the U.S. for the treatment of hemolytic anemia in adults with pyruvate-kinase, or PK, deficiency and in Europe for PK deficiency in adults.

 

Write to Colin Kellaher at colin.kellaher@wsj.com

 

(END) Dow Jones Newswires

October 17, 2025 08:36 ET (12:36 GMT)

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