FNIH Biomarkers Consortium Receives FDA Acceptance for Letter of Intent to Qualify Neurofilament as Biomarker of Rare Neurodegenerative Disease

North Bethesda, MD, May 16, 2024—The Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium has received acceptance from the U.S. Food and Drug Administration (FDA) for its letter of intent (LOI) to qualify neurofilament, a protein component of neurons, as a biological indicator of frontotemporal degeneration (FTD).

Launched in 2022, the “Neurofilament as a Fluid Biomarker of Neurodegeneration in Familial Frontotemporal Degeneration (FTD)” project seeks to advance the regulatory qualification of neurofilament as a biomarker to identify genetic carriers at risk of developing clinical FTD and enable their participation in clinical trials for new therapies. Submission of an LOI, which provides early information about the need for a proposed biomarker and its intended context of use in drug development, is the first step in the FDA’s Biomarker Qualification Program.

“The FDA’s acceptance of this LOI represents a significant milestone for the project,” said Dana Connors, Director of the FNIH Biomarkers Consortium. “It allows the team to move forward with developing a formal biomarker qualification plan for future studies of neurofilament.”

FTD is an umbrella term for progressive, neurodegenerative diseases impacting the frontal and temporal lobes of the brain. It is the most common form of dementia for people under age 60. While the main cause of FTD is unknown, many people with the disease accumulate abnormal proteins which clump together in brain cells called neurons. When a neuron is damaged or dies, the level of neurofilament protein increases in both cerebrospinal fluid and blood.

Recent studies indicate that this increased neurofilament in the blood is a promising marker of early stages of neurodegeneration. The ability to measure neurofilament has the potential to help identify when someone is at risk of developing symptoms, especially in people at genetic risk for rare neurogenerative diseases, such as FTD and amyotrophic lateral sclerosis (ALS).

“The families affected by familial FTD are urgently asking for prevention trials, so that potential treatments can be tested to see if they could delay or prevent disease onset,” said Penny Dacks, PhD, Senior Director of Scientific Initiatives at the Association for Frontotemporal Degeneration and Co-Chair of the Neuroscience Steering Committee of the FNIH Biomarkers Consortium. “This biomarker qualification initiative is an important step to help identify people likely to convert to clinical disease and to encourage investment in such preventative clinical trial designs.”

The process for biomarker qualification is a resource- and time-intensive endeavor, and one that the FNIH is uniquely suited to facilitate. The FNIH brings together the cross-disciplinary expertise and resources needed to generate sufficient evidence to support biomarker qualification, including the biological rationale, analytical performance, and the data supporting relationships between the biomarker and the clinical outcome of interest.

About the Biomarkers Consortium
The Foundation for the National Institutes of Health’s Biomarkers Consortium leads cross-sector efforts to validate and qualify biomarkers that accelerate the development of new therapeutics and health technologies. The core operations of the Biomarkers Consortium are supported through its contributing membership program, which includes the National Institutes of Health, the U.S. Food and Drug Administration, private industry, and not-for-profit organizations.

About the Foundation for the National Institutes of Health
The Foundation for the National Institutes of Health (FNIH) builds public-private partnerships that connect leading biomedical scientists at the National Institutes of Health (NIH) with their counterparts in life sciences companies, academia, patient organizations, foundations, and regulatory agencies (including the Food and Drug Administration and European Medicines Agency). Through team science, the FNIH solves complex health challenges and accelerates breakthroughs for patients, regardless of who they are or what health threats they face. The FNIH contributes to the development of new therapies, diagnostics, and potential cures; advances global health; and celebrates and helps train the next generations of scientists. Established by Congress in 1990 to support the mission of the NIH, the FNIH is a not-for-profit 501(c)(3) charitable organization. For more information about the FNIH, please visit fnih.org. Follow us on social media: LinkedIn, X, Facebook.