FDA offers Alzheimer’s guidance
pharmafile | February 11, 2013 | News story | Research and Development, Sales and Marketing | Alzheimer's, FDA, Roche, lilly
The FDA has issued guidance to help companies developing new treatments for patients with early-stage Alzheimer’s disease.
The US regulator’s proposal on Alzheimer’s, which is now out to public consultation for two months, focuses on tackling the condition before it becomes noticeable (or overt).
Last May the US Department of Health and Human Services recommended that trials should be conducted on at-risk individuals without symptoms, with a view to creating new measures to pick up Alzheimer’s as early as possible.
“The scientific community and the FDA believe that it is critical to identify and study patients with very early Alzheimer’s disease before there is too much irreversible injury to the brain,” said Russell Katz, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research.
“It is in this population that most researchers believe that new drugs have the best chance of providing meaningful benefit to patients,” he added.
Estimates suggest that as many as 115 million people worldwide could be affected by dementia by 2050, making it a significant issue for international health authorities.
While available treatments slow the progression of the disease – thus giving patients and carers a better quality of life – there is no cure.
The most common cause of dementia in older people, Alzheimer’s is a progressive brain disease that destroys memory and thinking skills, rendering patients eventually incapable of carrying out basic living tasks.
The FDA’s current stance is that treatments for overt dementia need to show both an effect on abnormal thinking and on how well patients function.
This makes sense, demonstrating that drugs benefit a patient’s thinking and have a clinically meaningful outcome, such as improvement or lack of decline in patients.
But the problem comes with early Alzheimer’s disease, because many patients exhibit no impairment of global functioning, which means it is difficult to assess changes in function – and therefore hard to tell whether a treatment is having a clinically significant effect.
The new FDA guidance will be of great interest to pharma, since ageing populations worldwide will lead to increases in the disease’s prevalence, which in turn means there is a major market opportunity.
Roche and Eli Lilly are among the companies setting the pace in this therapy area, providing drugs free of charge to US investigators conducting a worldwide trial this year.
The study is looking at three investigational compounds – two of which are from Lilly – designed to counter the effects of amyloid beta, the main ingredient of brain plaques found in Alzheimer’s patients, in different ways.
Chiming with the FDA’s new guidance, the idea is to prevent the loss of cognitive function in patients: 160 people with inherited mutations that cause early-onset forms of the disease will be studied to identify whether the drugs can improve Alzheimer’s biomarkers.
Adam Hill
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