RAPS recognizes that the current situation in Ukraine impacts our members and customers on many levels. If you are directly impacted by the current situation in the region and are challenged to meet your deadlines or obligations to RAPS, please reach out to raps@raps.org so that we can defer those challenges. Your health and safety are paramount to us.

Regulatory Focus™ > News Articles > 2 > EMA Shows Support for Early Biomarker Use in Developing Alzheimer’s Drugs

EMA Shows Support for Early Biomarker Use in Developing Alzheimer’s Drugs

Posted 28 February 2018 | By Ana Mulero 

EMA Shows Support for Early Biomarker Use in Developing Alzheimer’s Drugs

The European Medicines Agency (EMA) has revised its clinical studies guideline for developing medicines intended for Alzheimer’s disease (AD) treatment, encouraging industry to use biomarkers and backing their potential.

“As the biomarker field is evolving, the possibility to detect changes and progression in vivo, opens new regulatory scenarios including the possibility to intervene directly on the neuropathology before the appearance of symptoms,” EMA said in the revised guideline released on Wednesday.

EMA’s updates follow on the heels of similar changes made to the US Food and Drug Administration (FDA) guidance on developing drugs for AD –  released earlier this month  – including a revision of its previously stated position to consider a demonstrated effect on various biomarkers serving “as the basis for an accelerated approval.”

It is anticipated that the opening of new paths for AD therapies via new regulatory guidelines will be embraced by industry.

“Currently available medicines for Alzheimer’s disease only treat its symptoms,” EMA said in a statement. “However, a number of therapies under development are targeting the biological mechanism of the condition to try and modify the course of the disease.”

EMA pointed to biomarkers for neuroinflammation and blood or metabolic signatures as some examples of the activities already underway on new biomarkers.

A new section was added to the EMA guideline on the role and type of biomarkers in AD clinical trials. It recommends separating biomarkers according to their potential context of use, such as diagnostics, enrichment or prognostics. It cautions, however, biomarkers “for the most part still require validation for many of these particular purposes,” which falls outside the scope of the guidance.  

EMA said its new guideline also addresses the impact of additional diagnostic criteria, such as asymptomatic stages, on clinical trial design; factors to consider with trial outcome parameters, as well as efficacy and safety considerations for designs and analyses. The revised guideline is set to come into effect 1 September.

EMA guideline


© 2022 Regulatory Affairs Professionals Society.