European Commission Grants Orphan Drug Designation to Florbetaben (18F) for Diagnosis of ATTR Amyloidosis

• Florbetaben (18F) has been granted Orphan drug designation for the diagnosis of ATTR amyloidosis
• Florbetaben (18F) previously received orphan drug designation as a diagnostic agent for the management of amyloid light chain (AL) amyloidosis in both the EU and US
• The efficacy of florbetaben (18F) in diagnosing cardiac amyloidosis is being evaluated in an ongoing multi-center Phase 3 study

BERLIN, June 12, 2025 /PRNewswire/ -- Life Molecular Imaging GmbH (LMI) announces today that florbetaben (18F) has been granted orphan designation by the European Commission for the diagnosis of Transthyretin (ATTR) Amyloidosis.

Systemic amyloidosis is a rare group of complex diseases caused by protein misfolding and subsequent deposition in tissues, resulting in progressive organ damage. ATTR amyloidosis appears to be the most common form of amyloidosis, with incidence rates rising, likely due to prior underdiagnosis of the disease. Growing awareness of ATTR amyloidosis among cardiologists is improving recognition and diagnosis, allowing patients to access life-saving therapies.

Despite the observed rise in the prevalence and incidence of cardiac ATTR amyloidosis – and its high prevalence among patients with heart failure – the overall frequency in the general population remains below the threshold for orphan drug designation (five cases per ten thousand inhabitants of newly diagnosed patients). As a result, EMA granted orphan drug designation to florbetaben (18F) as a diagnostic agent for ATTR amyloidosis.

Florbetaben (18F) was initially developed and approved for the detection of neuritic amyloid plaques in the brains of patients with cognitive decline. Preliminary data suggest that florbetaben (18F) may also detect and quantify ATTR amyloid deposits in the heart and other organs using positron emission tomography (PET) imaging, thereby supporting diagnosis of the condition. The current multi-center Phase 3 trial (NCT05184088) aims to further validate florbetaben (18F)'s efficacy in the diagnosis of cardiac amyloidosis, including the ATTR subtype.

"The orphan drug designation for florbetaben (18F) will support our efforts to validate this tracer for the diagnosis of both AL and ATTR cardiac amyloidosis. With the approval of several new treatment options, especially for ATTR cardiac amyloidosis, fast and reliable diagnosis of the disease becomes even more important." said Andrew Stephens, MD, PhD, Chief Medical Officer of LMI.

About florbetaben (18F)
Neuraceq^® (florbetaben (18F)) is a radioactive diagnostic agent indicated for Positron Emission Tomography (PET) imaging of the brain to estimate β-amyloid neuritic plaque density in adult patients with cognitive impairment who are being evaluated for Alzheimer's Disease (AD) and other causes of cognitive decline. Neuraceq has been approved for routine clinical use in this indication by FDA, EMA and MHRA, and has local regulatory approval in other countries such as Canada, China, Japan, Korea, Switzerland, Taiwan. Florbetaben (18F) is currently under investigation as a targeted radiopharmaceutical for the detection of amyloid deposits in the heart and other organs of patients with cardiac and systemic amyloidosis of AL and ATTR type.

Most Common Adverse Reactions
In clinical trials including demented and non-demented subjects, the most frequently observed adverse drug reactions in 872 subjects with 1090 florbetaben (18F) administrations were injection/application site erythema (1.7%), injection site irritation (1.1%), and injection site pain (3.4%).

About Life Molecular Imaging (LMI)
Life Molecular Imaging GmbH, together with its affiliates in the UK and US (LMI) is a radiopharmaceutical company dedicated to developing and offering novel cutting-edge PET radiopharmaceuticals for imaging of neurodegenerative and cardiovascular diseases. The organization strives to be a leader in the molecular imaging field. Our mission is to pioneer innovative PET products that improve early detection and characterization of chronic and life-threatening diseases, leading to better therapeutic outcomes and improved quality of life. By advancing novel PET radiopharmaceuticals for molecular imaging, LMI is focusing on a key field of modern medicine. To learn more about LMI, please visit https://life-mi.com. LMI, a member of the Life Healthcare group of companies, is being acquired by Lantheus Holdings, Inc. For more information about the pending acquisition, please visit https://www.lifehealthcare.co.za/news-and-info-hub/latest-news/life-healthcare-proposed-disposal-of-lmi/.

For media queries
Brittany Hahn | Marketing Communications Manager | Life Molecular Imaging
Tel: +1.484.735.2840 | b.hahn@life-mi.com 

For scientific information 
Iris Hardewig | Clinical Development| Life Molecular Imaging
Tel. +49 151 14569896| i.hardewig@life-mi.com
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