New Guidance Released on Treatment for Early Alzheimer's
The FDA has recently released a draft guidance to assist sponsors in the development of treatments for sporadic Alzheimer's disease.
On March 12, 2024, the FDA released a preliminary guide for creating treatments for early-stage Alzheimer's disease (AD). This guidance document is designed to help sponsors who are in the clinical phases of developing drugs for the early stages of sporadic Alzheimer's disease, before dementia becomes apparent.
In the document, the FDA uses the term "early AD" to describe these stages, emphasizing that the disease is a continuous process. It is noted that patients in the late Stage 3 of early AD and those in the early Stage 4 of overt dementia may have similar clinical presentations. The agency anticipates that the document will foster discussions among various stakeholders, including the Office of Neuroscience in the Center for Drug Evaluation and Research, the Office of Therapeutic Products in the Center for Biologics Evaluation and Research, sponsors, the scientific community, and the public, as deemed necessary. The draft guidance has been updated from the previous version released in February 2018. It is currently open for comments until June 10, 2024.
Based on the guidance document, in previous times, enrollment in clinical trials for AD treatments relied on clinical criteria that outlined the advanced stages of AD. As a result, the study participants showed the cognitive changes commonly seen in clinically evident AD, along with the level of functional impairment associated with overt dementia. Given the context, treatments that were approved were based on it.
The FDA document highlights the use of biomarkers to capture the underlying changes in Alzheimer's disease and the inclusion of subjects in the early stages of the disease. It is crucial to focus on these efforts due to the potential for early intervention in the disease process of AD. This is possible because AD progresses slowly and specific pathophysiological changes occur well before any noticeable symptoms appear. The ultimate objective of intervening in the presymptomatic or early symptomatic stages of Alzheimer's disease is to slow down or even reverse the underlying pathological process before any noticeable clinical symptoms appear. It is important to grasp methods of evaluating treatment effectiveness in the early stages of the disease.
The document provides information on the diagnostic criteria and clinical staging of AD, which can be used to determine the disease stages and select participants for clinical trials. This document does not include information on clinical trials specifically targeting patients with AD who have developed overt dementia or any of the autosomal dominant forms of AD. It does not apply to the treatment of other forms of dementia either.
Industry has been focused on treatment options for AD. In September 2023, Roche and Ionis Pharmaceuticals agreed to collaborate on an early stage program involving an investigational therapeutic that targets RNA for the treatment of AD. Roche has obtained exclusive global rights and will handle the clinical development, manufacturing, and commercialization of the candidate on a global scale. The agreement expanded on a decade-long collaboration between the companies, which began in 2013. In 2018, the companies joined forces once again to work on a collaborative project. Their goal was to develop an Ionis therapeutic candidate for the treatment of immunoglobulin A nephropathy and geographic atrophy.
Through this new collaboration, Ionis aims to expedite the exploration and advancement of innovative treatments for individuals worldwide who are affected by Alzheimer's disease and Huntington's disease. By working together on these two programs, Ionis is able to make progress on our own initiatives in neurology that are in line with our strategic goals.
Through the expansion of their alliance, they are able to combine the collective expertise of the companies in the field of Alzheimer's disease and Huntington's disease with Roche's established track record in developing and bringing innovative neuroscience treatments to market.
Source: FDA; Ionis Pharmaceuticals
