Life Molecular Imaging (LMI) has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for its investigational PET radiopharmaceutical, [18F]florbetaben, in the diagnosis of cardiac amyloid light-chain (AL) and amyloid transthyretin-related (ATTR) amyloidosis. This designation aims to accelerate the development and review process for this potential diagnostic agent, addressing a significant unmet need in the early and accurate diagnosis of these serious conditions.
[18F]florbetaben, marketed as Neuraceq® for brain imaging, is already approved for detecting neuritic beta-amyloid plaques in the brain for Alzheimer's disease evaluation. The agent has also demonstrated the ability to identify amyloid deposits in the heart, leading to its Orphan Drug Designation in 2020 by both the European Commission and the FDA for diagnosing AL amyloidosis.
Clinical Significance
Cardiac amyloidosis occurs when amyloid proteins abnormally deposit in the heart, causing thickening and stiffening of the heart muscle. This can lead to heart failure, arrhythmias, and other life-threatening complications. Early and accurate diagnosis is crucial for timely intervention and improved patient outcomes. Current diagnostic methods can be complex and may involve invasive procedures such as biopsies.
Dr. Andrew Stephens, CMO of Life Molecular Imaging, stated, "Diagnosing cardiac AL amyloidosis is complex and challenging. [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."
Ongoing Phase 3 Trial
The efficacy of [18F]florbetaben in diagnosing cardiac amyloidosis is currently being evaluated in an open-label, multi-center Phase 3 trial (NCT05184088). This trial aims to visually and quantitatively assess PET imaging after administering 300 MBq of [18F]florbetaben to patients with suspected cardiac amyloidosis. The diagnostic efficacy will be compared to the standard of care diagnosis.
The trial includes patients 18 years or older with established systemic amyloidosis without proven cardiac involvement, known plasma cell dyscrasia, pathologically free light chain levels, or heart failure with preserved ejection fraction (EF). Key inclusion criteria also require either a mean left ventricular (LV) wall and septum thickness greater than 12 mm or NT-proBNP levels greater than 335 ng/L. The primary outcome measures the sensitivity and specificity of visual assessment of the imaging for diagnosing cardiac AL amyloidosis. Secondary endpoints include the sensitivity and specificity of quantitative assessments, correlation with LV EF and LV mass, correlation with AL markers, impact on patient management, and adverse event monitoring. The study is expected to be completed in June 2025.
Implications of Fast Track Designation
The FDA's Fast Track designation is designed to expedite the development and review of drugs and diagnostics that address serious conditions and unmet medical needs. This designation provides LMI with opportunities for more frequent interactions with the FDA review team, potentially leading to a faster approval process for [18F]florbetaben as a diagnostic tool for cardiac amyloidosis.
Dr. Ludger Dinkelborg, CEO of Life Molecular Imaging, commented, "The FDA's Fast Track Designation is a pivotal achievement that highlights the potential of [18F]Florbetaben to address a critical gap in diagnosing cardiac AL and ATTR amyloidosis."
