FDA Places Hold on Investigational Anemia Treatment

The FDA has placed a clinical hold on further investigation of AG-519 – an oral pyruvate kinase-R activator for the treatment of anemia – due to liver toxicity (cholestatic hepatitis) related to the 300 mg dose, according to the drug’s manufacturer (Agios Pharmaceuticals). The company considered moving forward with the drug at a lower dose, but decided to discontinue testing after patient follow-up and consultation with the FDA.

AG-519 is designed to activate pyruvate kinase and boost the cellular energy and life span of red blood cells. There are no approved drugs to treat the underlying cause of pyruvate kinase deficiency.

AG-519 was being assessed for safety, tolerability, pharmacokinetics, pharmacodynamics, and bioavailability in a phase I study of healthy volunteers in the United Kingdom. In addition, AG-519 was undergoing a palatability study in 98 volunteers in the United States to develop a formulation for potential future development; however, no volunteers or patients are currently receiving the drug.

Source: Agios Pharmaceuticals press release, December 15, 2016.