FDA Updates Label for Agios’ PYRUKYND with New Liver Injury Warning

FDA Label Update:
The FDA has approved a label update for Agios Pharmaceuticals' PYRUKYND (mitapivat) that includes a new warning about the risk of liver injury2 4.

Liver Injury Risk:
The updated label instructs prescribers to obtain liver tests prior to initiating PYRUKYND and monthly thereafter for the first six months and as clinically indicated. It advises interrupting or discontinuing PYRUKYND if clinically significant increases in liver tests are observed or if alanine aminotransferase is >5x ULN1 3.

Thalassemia Treatment:
PYRUKYND is being evaluated for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The FDA has accepted Agios' supplemental New Drug Application (sNDA) for this indication, with a PDUFA goal date of September 7, 20251 3.

Market Impact:
The disclosure of the new liver-related safety warning led to a significant drop in Agios' stock price, indicating market concern over the potential impact on the drug's adoption and sales5.