PRESS RELEASE

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, Germany, June, 12, 2025 – 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
UK-Neuraceq-25-00001

Neuraceq® - Product Indications And Use

PRODUCT INDICATIONS AND USE: Neuraceq is 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. A negative Neuraceq scan indicates sparse to no neuritic plaques and is inconsistent with a neuropathological diagnosis of AD at the time of image acquisition; a negative scan result reduces the likelihood that a patient’s cognitive impairment is due to AD. A positive Neuraceq scan indicates moderate to frequent amyloid neuritic plaques; neuropathological examination has shown this amount of amyloid neuritic plaque is present in patients with AD, but may also be present in patients with other types of neurologic conditions as well as older people with normal cognition. Neuraceq is an adjunct to other diagnostic evaluations.

Limitations: Limitations of Use
A positive Neuraceq scan does not establish the diagnosis of AD or any other cognitive disorder. The safety and effectiveness of Neuraceq have not been established for Predicting the development of dementia or other neurologic conditions or monitoring responses to therapies.

IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS: None

WARNINGS AND PRECAUTIONS

  • Risk for Image Misinterpretation and other Errors
    Errors may occur in the Neuraceq estimation of brain neuritic β-amyloid plaque density during image interpretation [see Clinical Studies (14)]. Image interpretation should be performed independently of the patient’s clinical information. The use of clinical information in the interpretation of Neuraceq images has not been evaluated and may lead to errors. Errors may also occur in cases with severe brain atrophy that limits the ability to distinguish gray and white matter on the Neuraceq scan. Errors may also occur due to motion artifacts that result in image distortion. Neuraceq scan results are indicative of the presence of brain neuritic β-amyloid plaques only at the time of image acquisition and a negative scan result does not preclude the development of brain neuritic β-amyloid plaques in the future.
  • Radiation Risk
    Neuraceq, similar to other radiopharmaceuticals, contributes to a patient's overall long-term cumulative radiation exposure. Long-term cumulative radiation exposure is associated with an increased risk of cancer. Ensure safe handling to protect patients and health care workers from unintentional radiation exposure [see Dosage and Administration.

ADVERSE REACTIONS:

  • The most commonly reported adverse reactions in clinical trials were injection site pain (3.4%), injection/appliucation site erythema (1.7%), injection site irritation (1.1%).

DRUG INTERACTIONS

  • Drug-drug interaction studies have not been performed in patients to establish the extent, if any, to which concomitant medications may alter Neuraceq image results.

USE IN SPECIFIC POPULATIONS

  • Pregnancy: All radiopharmaceuticals, including Neuraceq, have a potential to cause fetal harm depending on the stage of fetal development and the magnitude of the radiopharmaceutical dose. If considering Neuraceq administration to a pregnant woman, inform the patient about the potential for adverse pregnancy outcomes based on the radiation dose from the drug and the gestational timing of exposure.
  • Lactation: There are no data on the presence of florbetaben F 18 injection in human milk, the effects on the breastfed infant, or the effects of florbetaben F 18 injection on milk production. Exposure of Neuraceq to a breastfed infant can be minimized by temporary discontinuation of breastfeeding. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for Neuraceq and any potential adverse effects on the breastfed child from Neuraceq or from the underlying maternal condition.
  • Pediatric Use: Neuraceq is not indicated for use in pediatric patients.
  • Geriatric Use: No overall differences in safety were observed between older and younger subjects

OVERDOSAGE
A pharmacological overdose of Neuraceq is unlikely given the relatively low doses used for diagnostic purposes. In the event of administration of a radiation overdose with Neuraceq, the absorbed organ dose to the patient should be reduced by increasing elimination of the radionuclide from the body by inducing frequent micturition. Prior to Neuraceq administration, please read the full Prescribing Information for additional Important Safety Information.

SUSPECTED ADVERSE REACTIONS please report to: https://www.fda.gov/safety/medwatch-fda-safety-information-and-adverse-event-reporting-program

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