#Germany #Berlin #Life Molecular Imaging #Florbetaben #ATTR Amyloidosis

European Commission Grants Orphan Drug Designation to Florbetaben (18F)

Life Molecular Imaging GmbH (LMI) announced on June 12, 2025, that its novel imaging agent, florbetaben (18F), has received orphan drug designation from the European Commission specifically for diagnosing Transthyretin (ATTR) amyloidosis. This designation highlights the significance of this tracer in the diagnostic landscape of rare diseases, specifically given the increasing incidence of ATTR amyloidosis among patients with heart failure.

Systemic amyloidosis is categorized as a rare, complex disease brought about by the misfolding of proteins and their deposition within various tissues, which in turn leads to progressive damage to organs. While ATTR amyloidosis is recognized as one of the most prevalent forms within this group, its occurrence in the general population still remains lower than the threshold required for orphan drug status—set at five new patients per ten thousand inhabitants. This orphan designation is critical as it allows for more nuanced clinical insights and access to potential therapeutic avenues for affected patients.

The rise in the frequency of diagnosed cases of cardiac ATTR amyloidosis can be attributed to a heightened awareness among healthcare professionals, particularly cardiologists, thereby promoting earlier diagnosis and enabling timely treatment interventions. As more effective therapies become available, rapid and accurate diagnostics are essential to improve patient outcomes—an area florbetaben (18F) aims to address.

Florbetaben was originally developed to detect neuritic amyloid plaques in the brains of individuals exhibiting cognitive decline. What is noteworthy is the preliminary data suggesting that this imaging agent may also be effective in detecting and quantifying ATTR amyloid deposits not only in the heart but also in other organs using positron emission tomography (PET) imaging. An ongoing multi-center Phase 3 trial (NCT05184088) is currently exploring its efficacy for diagnosing cardiac amyloidosis, particularly the ATTR subtype.

Dr. Andrew Stephens, Chief Medical Officer at LMI, expressed enthusiasm, stating, "The orphan drug designation for florbetaben (18F) will facilitate validation of this tracer for diagnosing both AL and ATTR cardiac amyloidosis. With the advent of multiple new treatments for ATTR cardiac amyloidosis, streamlined and reliable diagnosis has never been more crucial."

About Florbetaben (18F)

Florbetaben (18F), marketed as Neuraceq®, is a radiopharmaceutical that utilizes PET imaging to gauge β-amyloid neuritic plaque density within the brains of adult patients suspected of having Alzheimer's Disease or other cognitive impairments. This compound has secured approvals in various regions—including the FDA, EMA, and MHRA—confirming its readiness for clinical use. Notably, florbetaben is under investigation as a targeted radiopharmaceutical for detecting amyloid deposits in the hearts of patients presenting with AL and ATTR amyloidosis.

Common Adverse Reactions

Clinical trials report a couple of adverse reactions associated with florbetaben (18F). The most frequent among 872 subjects administered the tracer were injection site erythema (1.7%), injection site irritation (1.1%), and injection site pain (3.4%). Monitoring these reactions is a part of the safety protocols as LMI furthers its investigations.

About Life Molecular Imaging

Life Molecular Imaging GmbH is a pioneering radiopharmaceutical company primarily engaged in the development of advanced PET radiopharmaceuticals aimed at imaging neurodegenerative and cardiovascular conditions. LMI’s objective is to lead advancements in molecular imaging, enhancing early disease detection and bettering therapeutic outcomes for patients. This commitment positions the organization at the frontier of modern medical innovations. For comprehensive details on their projects and news, please visit LMI's website. LMI is currently in the process of being acquired by Lantheus Holdings, Inc., a further step in solidifying its role in this critical healthcare space.

For any media inquiries, please contact:
Brittany Hahn | Marketing Communications Manager | Life Molecular Imaging
Tel: +1.484.735.2840
Email: [email protected]

We are at an exciting crossroads in the realm of amyloidosis diagnosis, and LMI’s contributions through florbetaben (18F) exemplify the ongoing evolution in patient care and treatment efficacy.