A lack of clarity about requirements for cardiovascular safety and outcomes data in updated EMA guidance means companies developing lipid-modulating therapies will need to seek individual advice from the European regulator as early as possible in development.

Although companies developing LDL-lowering molecules still can use LDL reduction as a primary endpoint, the new guidance does nothing to clear up ambiguity in 2004 guidelines about what the threshold is for ruling out a detrimental effect on CV morbidity and mortality.