Lipid advice in Europe
Why companies will need EMA advice on CV requirements for lipid disorders
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.
For non-LDL-lowering therapies - such as compounds designed to lower triglycerides or raise HDL - the new guidance is more stringent in that it now requires companies to demonstrate a CV outcomes benefit.
Moreover, the agency says it will determine case-by-case whether those data are required before or after approval.
Cardiovascular outcomes data are not expected to be required for statins because there is enough evidence that they reduce CV events.
Under EMA's 2004 guidance on the development of therapies for lipid disorders, a