Alzheimer’s Disease: Companies Weigh Revised FDA Draft Guidance

Regulatory NewsRegulatory News | 21 May 2018 |  By 

Companies developing treatments for early Alzheimer’s Disease submitted another round of positive comments last week on US Food and Drug Administration (FDA) draft guidance, offering a hint of optimism in a space where no company has seen success.

With setbacks and failures in the development of Alzheimer’s treatments from Merck, Eli Lilly, AstraZeneca and others, it should come as no surprise that companies are interested in this latest revised draft and happy that the agency is open to using the accelerated approval pathway for at least some future Alzheimer’s development programs.

The 10-page draft, released in mid-February as part of a suite of guidance documents on neurological disorder treatments, revises a previous draft from 2013 and offers sections on diagnostic criteria, outcome measures, including clinical endpoints for early AD trials in Stage 3, 2 and 1 patients.


While praising FDA for recognizing “the evolution in the field of AD research and drug development,” Lilly called for the possibility of “full approval (for inherently meaningful cognitive effects in Stage 2) or accelerated approval with post-approval requirement (for cognitive effects in Stage 2 and biomarker effects in Stage 1)” to “allow efficacious drugs to be available for clinical use more quickly.”

Lilly noted ongoing Phase 3 studies with combined Stage 3 and 4 populations from Roche, Biogen, Lilly and AstraZeneca, and Eisai and Biogen.

Denali Therapeutics, which recently closed a $155 million deal with Takeda to develop treatments for neurodegenerative diseases, called on FDA to provide more concrete examples that meet the criteria for general outcome measures that are acceptable for clinical trials.

“More detailed examples would help determine the appropriate assessment tools to use in clinical trials (e.g. lines 198-200, examples of neuropsychological tests in which a meaningful change would support marketing approval in Stage 2 patients),” Denali said.

Biogen also applauded FDA’s “openness to exploring the accelerated approval pathway for future AD programs. Biogen recognizes that at this time there may not be sufficient evidence that an observed treatment effect on biomarker measures is reasonably likely to predict clinical benefit. However, the Agency’s acknowledgement that this pathway may apply, as scientific understanding progresses, will foster critical dialogue between sponsors, the Agency and the broader scientific community.”

Roche, AstraZeneca, BioClinica and others also sized up the revised draft with comments this month.



© 2022 Regulatory Affairs Professionals Society.

Discover more of what matters to you

No taxonomy