The FDA’s acceptance of the LOI submitted by the NIMBLE team signals the regulatory agency’s support of the project’s proposed path forward and its invitation to continue the process of qualifying biomarkers.

An estimated 16.5 million people in the United States are living with NASH, a disease associated with obesity and diabetes and characterized by buildup of fatty tissue in the liver. NASH does not present any symptoms until it progresses to later stages, at which point the liver may already have incurred serious damage, including cirrhosis, potentially requiring a liver transplant to prevent cancer or death. Currently, the only way to definitively diagnose a patient with NASH is through an extremely invasive and painful liver biopsy. Recent FDA guidance on this growing problem—soon to be the leading cause of liver transplant in the country—recommends the qualification of non-invasive biomarkers to replace liver biopsy for diagnosis.

The FDA’s qualification of a biomarker represents the agency’s conclusion that the biomarker is well-supported by evidence and may reliably be applied within a specific context of use in drug development. The FDA’s three-step approval process for qualifying new biomarkers begins with submission of an LOI, which provides some early information about the proposed biomarkers and their intended measurement and use. After FDA acceptance of the LOI, a qualification plan may be submitted, providing a more granular description of the steps the requestor will take to provide the necessary supportive data for qualification, such as a plan to address known knowledge gaps. Upon approval at this stage, the requestor may submit a full qualification package, putting forth all the evidence the FDA needs to make its decision to qualify the new biomarkers. If the provided data support qualification within the specified context of use, the FDA will then qualify the biomarkers for anyone to use in the development of new therapies.

The FDA’s acceptance of the NIMBLE team’s LOI is a major milestone for the project, which ultimately aims to enable simpler diagnosis and better treatment for people with this silent and pervasive liver disease.

For more information about the project, click here.