Life Molecular Imaging (LMI) announced on January 21, 2025, that the U.S. Food and Drug Administration has granted Fast Track Designation to [18F]florbetaben for diagnosing cardiac amyloid light-chain (AL) and amyloid transthyretin-related (ATTR) amyloidosis. This designation is designed to facilitate development and expedite evaluation of drugs treating serious conditions with unmet medical needs.
Expanding Beyond Brain Imaging
[18F]florbetaben is currently validated and approved for detecting neuritic beta amyloid plaques in the brain, marketed globally as Neuraceq®. The compound has demonstrated its capability to identify amyloid deposits in the heart, including both AL and ATTR types. These findings previously led to Orphan Drug Designation by both the European Commission and FDA in 2020 specifically for AL amyloidosis diagnosis.
"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," said Dr. Ludger Dinkelborg, CEO of Life Molecular Imaging.
Addressing Diagnostic Challenges
The complexity of diagnosing cardiac AL amyloidosis represents a significant clinical challenge. Dr. Andrew Stephens, MD, PhD, CMO of Life Molecular Imaging, explained that "[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."
Current Clinical Development
A Phase 3 trial (NCT05184088) is currently underway to further validate [18F]florbetaben's efficacy in cardiac amyloidosis diagnosis. The compound is being investigated as a targeted radiopharmaceutical for detecting amyloid deposits in the heart and other organs of patients with cardiac and systemic amyloidosis of AL and ATTR type.
Safety Profile
In clinical trials involving 872 subjects with 1,090 [18F]florbetaben administrations, the most frequently observed adverse reactions were injection/application site erythema (1.7%), injection site irritation (1.1%), and injection site pain (3.4%).
Regulatory Approvals
Neuraceq® has received approval for routine clinical use in brain amyloid imaging from the FDA, EMA, and MHRA, with additional local regulatory approvals in countries including Canada, UK, Japan, China, Taiwan, and Korea.
