Biomarker Qualification Submissions
The table below lists information about submissions to the FDA biomarker qualification program for which final biomarker qualification decisions have not yet been made. This table includes legacy projects (those submitted prior to passage of the 21st Century Cures Act, Section 3011 legislation [Food, Drug and Cosmetic Act (FD&CA), new section 507 process or “section 507 process”]) as well as those submitted as part of the 507 process. This table is updated on a biannual basis and provides information on the biomarker qualification project, FDA’s decision to Accept or Not Accept the submission, and FDA’s recommendations on further biomarker development.
*Information submitted to FDA by outside parties requesting qualification of a drug development tool is not endorsed or recommended by FDA. Submissions are made publicly available in accordance with the 21st Century Cures Act. The FDA makes no representations, guarantees, or warranties as to the accuracy, completeness, currency, or suitability of the information in submissions. See the FDA decision letter corresponding to each submission for FDA considerations and recommendations related to each request for qualification.
For more information about the Biomarker Qualification Program, you may contact:
CDER-BiomarkerQualificationProgram@fda.hhs.gov.
Biomarker Qualification (BQ) Submissions
| Requestor | Abbreviated Biomarker Description | Abbreviated COU | * Qualification Submission & Reviewable Date | FDA Submission Decision & Recommendations |
|---|---|---|---|---|
|
Menarini Silicon Biosystems Inc. / Memorial Sloan Kettering Cancer Center (MSKCC) |
The biomarker “CTC0” is a single factor biomarker that registers the change in circulating tumor cell (CTC) numbers between two time points, baseline and after 12 weeks of therapy. |
CTC0 is a Response Biomarker for the early evaluation of drug product activity in castration resistant prostate cancer (mCRPC) clinical trials. |
507 Update Response 08/12/2020 |
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|
Critical Path Institute (C-Path), |
Skeletal muscle injury (SKM1-8) biomarkers as assessed by immunoassay |
Safety biomarkers to monitor skeletal muscle degeneration in rats with serum creatinine kinase and serum aspartate aminotransferase (AST) |
Legacy project in transition to 507 process |
|
|
International Life Sciences Institute (ILSI) /Health and Environmental Sciences Institute (HESI) |
Transcriptomic biomarker panel as assessed by RT-qPCR and microarray |
Safety biomarker to identify in-vitro mammalian cell structural chromosomal damage |
Legacy project in transition to 507 process |
|
|
Joint Qualification Committee of the Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium (BC) and the Radiologic Society of North America (RSNA) Quantitative Imaging Biomarker Alliance (QIBA) |
Standardized uptake value (SUV) of tumors as measured by quantitative FDG-PET/CT |
Pharmacodynamic/response biomarker to accelerate evaluations of novel treatment of NSCLC (lung cancer) and DLBCL (lymphoma) |
Legacy project in transition to 507 process |
|
|
FNIH/BC- RSNA/QIBA |
Tumor Volume as measured by CT |
Pharmacodynamic/response biomarker used to predict disease progression or tumor response in primary and metastatic cancers |
Legacy project in transition to 507 process |
|
|
C-Path PSTC Nephrotoxicity Working Group (NWG) |
Urinary biomarkers of kidney injury as assessed by immunoassay |
Safety biomarkers to complement the detection of acute drug-induced renal tubule injury in rat |
Legacy project in transition to 507 process |
|
|
C-Path/CAMD |
Hippocampal volume as measured by MRI |
Prognostic biomarker to enrich studies of amnestic mild cognitive impairment patients who are likely to develop progressive Alzheimer's disease |
Legacy project in transition to 507 process LOI Withdrawn |
|
|
FLUIDDA, Inc. |
Lung and airway structural and functional parameters as assessed by CT |
Pharmacodynamic/response biomarker to assess response to an intervention in COPD and asthma patients |
Legacy project in transition to 507 process |
|
|
Safer And Faster Evidence-based Translation (SAFE-T) Consortium |
Urine and serum biomarkers of drug-induced kidney injury as assessed by assay |
Safety biomarkers to detect drug-induced kidney injury |
Legacy project in transition to 507 process |
|
|
SAFE-T Consortium |
Serum biomarkers of drug-induced liver Injury as assessed by multiple assays |
Safety biomarkers to detect drug-induced hepatic injury o:p> |
Legacy project in transition to 507 process |
|
|
SAFE-T Consortium |
Serum biomarkers of drug-induced vascular injury as assessed by multiple assays |
Safety biomarkers to detect a risk of vascular injury |
Legacy project in transition to 507 process |
|
|
AnaBios Corporation |
Algorithm-derived biomarker as assessed by myocardial cell assay |
Safety biomarker to rank pro-arrhythmic drugs based on likelihood of arrhythmia |
Legacy project in transition to 507 process |
|
|
Fossa Consulting |
Beat to beat restitution as assessed by ECG |
Safety biomarker for arrhythmia risk |
Legacy project in transition to 507 process LOI Withdrawn |
|
|
Drs. Benesic and Gerbes |
Proteomic biomarker panel as assessed from blood monocytes using a cell-based assay |
Safety biomarker to assess idiopathic drug-induced liver injury |
Legacy project in transition to 507 process |
|
|
C-PATH PSTC Hepatotoxicity Working Group (HWG)
Contact: Elizabeth Walker |
Serum Glutamate Dehydrogenase (GLDH) as assessed by immunoassay |
Safety biomarker to detect drug-induced liver injury |
Legacy project in transition to 507 process |
|
|
Perspectum Diagnostics |
Iron corrected liver inflammation and fibrosis (LIF) biomarkers as assessed by MRI |
Prognostic biomarker to identify patients who are more likely to have liver histopathology for nonalcoholic steatohepatitis (NASH) |
Legacy project in transition to 507 process |
|
|
C-PATH MREC |
Anatomic features of the terminal ileum and large bowel assessed by MRI |
Pharmacodynamic/response biomarker for Crohn’s disease used as a co-primary endpoint |
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|
COPD Foundation |
Blood Eosinophil count as assessed by blood analyzer |
1. Predictive biomarker for subjects who are more likely to exhibit COPD exacerbations |
1. LOI - Not Accepted 12/18/2017 |
|
|
CPATH Contact: Martha Brumfield |
Lipoarabinomannan as assessed by immunoassay |
Pharmacodynamic response biomarker to assess treatment response in patients with pulmonary tuberculosis |
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|
CPATH Contact: Inish O’Doherty |
Islet cell autoantibodies as assessed by immunoassay |
Susceptibility/risk biomarker to identify individuals more likely to develop type 1 diabetes |
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Center for Studies of Addiction: University of Pennsylvania Perelman School of Medicine Contact: H. Kranzler, M.D. |
rs678849, a SNP in ORPD1, as identified by genotyping assay | Predictive biomarker for certain subjects opioid use disorder to enrich buprenorphine trials. | LOI 6/11/2018 |
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|
CPATH Critical Path Institute's Predictive Safety Testing Consortium Nephrotoxicity Working Group (CPATH PSTC-NWG), and Foundation for the National Institutes of Health’s Biomarker Consortium Kidney Safety Biomarker Project Team (FNIH BC-KSP) |
Urinary nephrotoxicity biomarkers as assessed by immunoassays |
Safety biomarker panel to assess whether a drug has caused mild injury response in the renal tubules in normal healthy volunteers and patients with normal renal function |
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|
The Progeria Research Foundation Contact: Leslie Gordon, M.D., Ph.D. |
Progerin, an abnormal splice variant of the inner nuclear membrane protein lamin A | Pharmacodynamic/response biomarker to assess drug intervention in future clinical treatment trials of Progeria | LOI 6/27/2018 |
LOI - Accepted 9/5/2018 |
|
Critical Path Institute (CPATH) Contact: John-Michael Sauer, Ph.D. |
Glutamate Dehydrogenase (GLDH) | Safety biomarker for drug induced liver injury in healthy subjects and patients in all stages of drug development trials | 507 Update 6/29/2018 |
507 Update Response 10/25/2018 |
|
Foundation for the National Institutes of Health (FNIH) Contact: Tania Nayak Kamphaus |
Proportional change in dual-energy x-ray absorptiometry (DXA) bone mineral density (BMD) | Surrogate endpoint for the clinical endpoints of hip and non-vertebral fracture risk reduction | 507 Update 8/25/2018 |
|
|
Nerve Unit Massachusetts General Hospital Contact: Ann L. Oaklander |
Epidermal Neurite Density as manually counted at the dermal-epidermal junction |
Diagnostic biomarker, used with other clinical indicators, to confirm a diagnosis of SFPN in drug development studies
|
|
|
| Rady Faculty of Health Sciences University of Manitoba Contact: Peter L. Nickerson |
HLA DR/DQ Eplet Mismatch Score manually counted from software mismatch identification | Prognostic biomarker, used with other testing, to categorize kidney transplant recipients’ risk of graft rejection for enrichment or stratification in drug development studies | LOI 8/13/2018 |
LOI - Accepted 12/4//2018 |
| Perspectum Diagnostics Ltd Contact: Dr. Jaco Jacobs |
Iron Corrected T1 (cT1) MR image of liver tissue | Diagnostic enrichment biomarker used in conjunction with clinical risk factors to identify patients more likely to have liver histopathologic findings of Nonalcoholic steatohepatitis (NASH) | 507 Update 10/16/2018 |
507 Update Response 1/19/2019 |
|
FLUIDDA, Inc. Contact: Jan De Backer |
Volume of lower lung lobes (VLLL) as measured by CT | Monitoring biomarker, used with other parameters, for correlation with changes in IPF status in drug development studies | LOI 8/13/2018 |
LOI - Not Accepted 1/29/2019 |
|
Biomarkers Consortium, Foundation for the National Institutes of Health (FNIH) & The Radiologic Society of North America, Quantitative Imaging Biomarkers Alliance (RSNA‐QIBA) Contact: Dr. Linda Doody, PhD |
Tumor volume change as measured by CT | Pharmacodynamic/Response biomarker to assess tumor volume change for new oncologic drug clinical trial therapy of solid tumors | 507 Update 11/2/2018 |
507 Update Response 2/19/2019 |
|
Perspectum Diagnostics Contact: Jaco Jacobs, PhD |
MRI Measured Proton Density Fat Fraction (MRI-PDFF) of Liver | Diagnostic enrichment biomarker for selecting patients for non-alcoholic steatohepatitis (NASH) trials | LOI 12/2/2018 |
LOI - Accepted 4/12/2019 |
|
SAFE-T |
Drug-induced vascular injury (DIVI) biomarkers measured in the blood by immunoassays and mass spectrometry |
Monitoring biomarker for DIVI, used with other parameters, to measure inflammation, vascular endothelial and smooth muscle damage. |
507 Update 01/24/2019 |
|
|
AnaBios Corporation Contact: Dr. Jack A. Reynolds |
The safety biomarker for preclinically identifying the pro-arrhythmia risk of drugs, with specific focus on Torsade de Pointes (TdP) type arrhythmias | The Pro-Arrhythmia Score will be used at the preclinical stage of drug discovery to assess the TdP pro-arrhythmia risk of a test article, in order to facilitate clinical development and risk management planning, and support benefit-risk analysis, as well as enable regulatory decision making. | 507 Update 4/16/2019 |
507 Update Response 5/6/2019 |
|
FNIH Biomarkers Consortium Contact James McPartland, PhD |
N170 to Upright Faces | Diagnostic biomarker to enrich clinical trials by reduction of ASD-associated heterogeneity. | LOI 1/31/2019 |
LOI - Accepted 5/6/2019 |
|
Foundation for the Contact: J. Menetski, PhD |
FNIH-BC Progress OA Project Biomarkers |
Prognostic biomarkers to identify individuals more likely to experience knee osteoarthritis disease progression | 507 Update 1/21/2019 |
507 Update Response 5/6/2019 |
|
Critical Path Institute (CPATH)Predictive Safety |
Glutamate Dehydrogenase (GLDH) |
Safety biomarker for drug induced liver injury in healthy subjects and patients in all stages of drug development trials |
QP 11/14/19 |
|
|
Critical Path Institute (CPATH)Predictive Safety Testing Consortium |
Skeletal Muscle Injury Biomarker Panel |
Safety biomarker panel biomarker to aid in the detection of acute drug induced skeletal muscle injury in phase 1 trials |
LOI 12/20/2019 |
|
|
Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium |
Four circulating biomarker panels (NIS-4, OWL, PRO-C3-PRO-C6, ELF; alone or in combination) for NASH |
Diagnostic enrichment biomarker intended for use, in conjunction with clinical factors, to identify patients likely to have liver histopathologic findings of nonalcoholic steatohepatitis (NASH) and with a nonalcoholic fatty liver disease activity score (NAS) ≥4 and liver fibrosis stages 2 or 3 (by Brunt/Kleiner scale) |
LOI 10/29/2019 |
|
|
Tufts Medical Center |
End Stage Knee Osteoarthritis (esKOA) Score |
Prognostic biomarker to identify patients likely to experience long-term disease progression. |
LOI 9/05/2019 |
LOI-Accepted 12/20/2019 |
|
Tufts Medical Center |
Cumulative Damage and Disease Activity Scores |
Prognostic enrichment biomarker to identify patients likely to experience long-term disease progression |
LOI 9/5/2019 |
LOI-Accepted 12/20/2019 |
|
Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium |
Trabecular bone texture (TBT) biomarkers (n=six) from fractal signature analysis (FSA) curves generated from the tibial subchondral region of plain knee radiographs; the six biomarkers are the vertical filter (VF) intercept, VF linear slope, VF quadratic slope, horizontal filter (HF) intercept, HF linear slope, and HF quadratic slope. |
Prognostic enrichment imaging biomarker panel for use in phase 2 and 3 clinical trials to identify individuals with a diagnosis of knee osteoarthritis who are likely to experience disease progression within the subsequent 48 months based on the WOMAC pain subscale and/or radiographic joint space width loss and/or joint replacement. |
LOI 9/19/2019 |
LOI-Accepted 1/21/2020 |
|
Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium |
Osteoarthritis prognostic biomarkers as assessed by immunoassays. |
Prognostic enrichment molecular biomarkers for use in phase 2 and 3 clinical trials to identify individuals with a diagnosis of knee osteoarthritis who are likely to experience disease progression within the subsequent 48 months based on the WOMAC pain subscale and/or radiographic joint space width loss and/or joint replacement. |
LOI 9/18/2019 |
LOI-Accepted 2/25/2020 |
|
Foundation for the National Institutes of Health Autism Biomarkers Consortium for Clinical Trials (FNIH ABC-CT) |
Oculomotor Index of Gaze to Human Faces |
Diagnostic biomarker to be used with clinical and demographic characteristics to select a less heterogeneous subgroup within subjects with autism spectrum disorder (ASD) for clinical trial enrichment. |
LOI 1/30/2020 |
LOI-Accepted 3/17/2020 |
|
TransBioLine |
Drug-induced vascular injury (DIVI) biomarkers measured in the blood by immunoassays and mass spectrometry |
Monitoring biomarker for DIVI, used with other parameters, to measure inflammation, vascular endothelial and smooth muscle damage. |
LOI 11/12/2019 |
|
|
LITMUS |
Composite biomarker consisting of PRO-C3 and FAST Score (Liver Stiffness Measurement, Controlled Attenuation Parameter, and aspartate aminotransferase (AST)) |
Diagnostic enrichment biomarker intended for use, in conjunction with clinical factors, to identify patients likely to have liver histopathologic findings of nonalcoholic steatohepatitis (NASH) and with a nonalcoholic fatty liver disease activity score (NAS) ≥4 and liver fibrosis stages 2 or 3 (by Brunt/Kleiner scale). |
LOI 12/18/2019 |
|
|
Critical Path Institute (C-Path), Transplant Therapeutics Consortium (TTC) |
Composite biomarker panel score to predict five-year risk of kidney allograft loss |
Reasonably likely surrogate endpoint, used in the first year after transplantation, to predict the five-year risk of allograft loss in kidney transplant patients for use in clinical trials to support evaluation of immunosuppressive therapy applications. |
LOI 2/14/2020 |
|
|
Resoundant, Inc. |
Magnitude of the complex shear modulus (|G*|) |
Diagnostic biomarker to pre-screen patients with clinical risk factors for chronic liver disease for enrolment in clinical trials to identify those at high risk of having histopathologic findings of significant fibrosis (≥F2), advanced fibrosis (≥F3), or cirrhosis (F4) on liver biopsy. |
LOI 5/4/2020 |
|
|
University of Washington Department of Laboratory Medicine |
Plasmodium 18S rRNA/rDNA |
Monitoring biomarker informs initiation of treatment with anti-malarial drug following controlled human malaria infection (CHMI) with P. falciparum sporozoites in healthy subjects from endemic areas in clinical studies for vaccine and/or drug development. |
LOI 3/10/2020 |
|
|
Innovative Medicines Initiative (IMI) TransBioLine Drug-Induced CNS Injury (DINI) Work Package |
Drug-induced serum-based CNS injury biomarker panel |
Safety biomarker panel to detect acute drug-induced central nervous system (CNS) injury risk in Phase 1 trials |
LOI 3/18/2020 |
|
|
Yale University |
Individualized Risk Calculator for Psychosis (IRC-P) |
Prognostic biomarker intended for use in clinical trials to enrich for individuals most likely to progress to full psychosis and poor long-term functional outcomes. |
LOI 4/6/2020 |
|
|
IGEA Research Corporation |
Free copper in serum assessed by inductively coupled plasma mass spectrometry (ICPMS) |
Serum Free Copper as a prognostic biomarker for conversion from mild cognitive impairment (MCI) to symptomatic Alzheimer’s disease (AD) typified by copper imbalance. |
LOI 6/11/2020 |
|
|
PathAI, Inc. |
The features comprising the Non-Alcoholic Fatty Liver Disease Activity Score (NAS) (the grade of steatosis, hepatocellular ballooning, inflammation) and fibrosis staging (via NASH/CRN Brunt/Kleiner scale), assessed on liver biopsy as interpreted by Artificial Intelligence (AI). |
Diagnostic biomarker to assess disease activity score components (i.e., steatosis, ballooning, inflammation) and fibrosis stage in liver biopsies as part of evaluation for enrollment in non-alcoholic steatohepatitis (NASH) clinical trials. |
LOI 6/8/2020 |
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|
University of Washington Department of Laboratory Medicine |
Plasmodium 18S rRNA/rDNA |
Biomarker endpoint to be used in clinical trials to evaluate drugs and/or vaccines intended to treat or prevent Plasmodium falciparum in endemic areas. |
LOI 5/18/20 |
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Innovative Medicines Initiative TransBioLine |
Drug-induced kidney injury biomarker panel |
Safety biomarker panel to detect acute drug-induced kidney injury risk in Phase 1 trials |
LOI 5/15/20 |
Additional Biomarker Information
- List of Qualified Biomarkers
- Resources for Biomarker Requestors
- Biomarker Qualification Program Submission FAQs
- Guidance and Process
- More About Biomarkers and Qualification
- 21st Century Cures Act: Qualification of Drug Development Tools
- Biomarker Qualification Program
Contact us at: CDER-BiomarkerQualificationProgram@fda.hhs.gov