FDA Grants Fast Track Designation to [18F]Florbetaben for Diagnosis of Cardiac Amyloidosis

The FDA granted fast track designation to [18F]florbetaben (Life Molecular Imaging) for the diagnosis of cardiac amyloid light-chain (AL) and amyloid transthyretin-related (ATTR) amyloidosis.¹

The FDA grants fast track designation to new diagnostic for cardiac amyloidosis. | Image Credit: Siarhei | stock.adobe.com

"Diagnosing cardiac AL amyloidosis is complex and challenging," Andrew Stephens, MD, PhD, chief medical officer of Life Molecular Imaging, said in a news release. "[18F]florbetaben PET imaging of patients with suspected cardiac amyloidosis has the potential to streamline the diagnostic process, providing earlier and appropriate access to therapy and improved monitoring of such interventions."¹

[18F]florbetaben PET, marketed as Neuraceq, is approved to detect neuritic beta amyloid plaque in the brain and has also demonstrated the ability to identify amyloid deposits in the heart. Previously, the diagnostic received orphan drug designation from both the European Commission and FDA in 2020 for the diagnostic of AL amyloidosis, according to the news release.¹

Amyloidosis is described as diseases caused when amyloid abnormally deposits into 1 or more organs. When deposited in the heart, it can cause the muscles to thicken and stiffen, which makes the heart weaker over time, leading to congestive heart failure, heart rhythm abnormalities, and valvular disease. Further, AL and ATTR can cause noncardiac symptoms, including carpal tunnel syndrome, lumbar spinal stenosis, peripheral and autonomic neuropathy, orthostatic hypotension poor appetite, diarrhea, and more. Currently, diagnostics can include blood work, imaging studies, such as echocardiogram, cardiac MRI, EKG, tissue biopsy, bone marrow biopsy, nerve conduction study, and genetic testing, according to Johns Hopkins Medicine.²

[18F]florbetaben is currently undergoing an open-label multicenter phase 3 trial, which will visually and quantitatively assess PET imaging after an application of 300 MBq of the study diagnosis and scanning of patients with suspected cardiac amyloidosis, according to the clinical trial information. The diagnostic efficacy will be compared to the standard of truth obtained through the standard of care diagnosis.³

Investigators included patients 18 years or older with 1 of the following: established systemic amyloidosis without proven cardiac involvement, known plasma cell dyscrasia, pathologically free light chain levels in serum or urine, or presence of heart failure with preserved ejection fraction (EF). Patients also had to have either mean left ventricular (LV) wall and septum thickness greater than 12 mm or NT-proBNP greater than 335 ng/L. Patients with known allergic reactions or hypersensitivity to the compound or the study drug; those who had severe hepatic impairment; those who were pregnant, lactating, or breastfeeding; and individuals unable to lay flat for up to 60 minutes were excluded.³

The primary outcome of the study included sensitivity and specificity of the visual assessment of the imaging for the diagnosis of cardiac AL amyloidosis. Secondary end points included sensitivity and specificity of the study drug for diagnostic of cardiac AL amyloidosis using quantifications, correlation of quantitative results with LV EF and LV mass, correlation of quantitative results with AL based on FLC-diff, cTnT, and NT-proBNP levels, impact of PET imaging on diagnostic of thinking and patient management via physician’s questionnaires prior to and after diagnostic work-up, and number of adverse events. Furthermore, other outcomes included the sensitivity and specificity of the images for a differential diagnosis between AL, ATTR, and non-cardiac amyloidosis.³

The study is currently in the recruiting stage with an estimated completion of June 2025.³

REFERENCES

1. Life Molecular Imaging Secures FDA Fast Track Designation for [18F]Florbetaben in Diagnosing Cardiac AL and ATTR Amyloidosis. News release. Life Molecular Imaging. January 21, 2024. Accessed January 22, 2025. https://prnmedia.prnewswire.com/news-releases/life-molecular-imaging-secures-fda-fast-track-designation-for-18fflorbetaben-in-diagnosing-cardiac-al-and-attr-amyloidosis-302356383.html

2. Johns Hopkins Medicine. Cardiac Amyloidosis. Accessed January 22, 2025. https://www.hopkinsmedicine.org/health/conditions-and-diseases/cardiac-amyloidosis

3. Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis (CArdiag). ClinicalTrials.gov ID: NCT05184088. Updated April 12, 2024. Accessed January 22, 2025. https://clinicaltrials.gov/study/NCT05184088