FDA slaps a hold on Agios’ PK drug, forc­ing the biotech to kill it in fa­vor of an in­ter­nal ri­val

A few days ago, Agios $AGIO was try­ing to de­cide whether it should take AG-348 or AG-519 in­to a piv­otal tri­al for rare cas­es of pyru­vate ki­nase (PK) de­fi­cien­cy. But now the FDA has made the de­ci­sion for Agios, hand­ing down a clin­i­cal hold on AG-519 that per­suad­ed the biotech to scrap the pro­gram al­to­geth­er.

The hold, Agios CEO David Schenkein tells me, was trig­gered by an ear­li­er case of cholesta­t­ic he­pati­tis that was re­port­ed at ASH a lit­tle more than a week ago. One of the pa­tients in a bioavail­abil­i­ty study tak­ing a 300 mg dose was hit by the liv­er dis­ease. And even though “she’s im­prov­ing” while Agios was al­so “like­ly to move to 50 mg or low­er (for the piv­otal), one can’t be sure we wouldn’t have seen this again.”

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