The European Commission has granted orphan drug designation to florbetaben (18F) for the diagnosis of transthyretin amyloidosis, including transthyretin amyloid cardiomyopathy (ATTR-CM), Life Molecular Imaging (LMI) announced in a recent press release.
“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 Chief Medical Officer Dr. Andrew Stephens.
Florbetaben (18F), marketed as Neuraceq®, is a radioactive tracer designed for use in positron emission tomography (PET) imaging. The tracer was originally approved in the United States, European Union and United Kingdom for the identification of amyloid plaques in the brains of patients with Alzheimer disease and other forms of cognitive decline.
What are amyloid fibrils?
In ATTR-CM, amyloid fibrils are clumps of faulty, misfolded TTR proteins that are deposited in the heart, nerves and internal organs. In the heart, fibrils affect the left ventricle, causing the heart tissue to thicken and stiffen. This makes it more difficult for the heart to pump blood around the body and can lead to heart failure.
Recent data have found that florbetaben (18F) may be used during PET scans to identify an accumulation of misfolded transthyretin in the heart, supporting its use in patients suspected to have ATTR-CM.
Read more about ATTR-CM testing and diagnosis
Orphan drug designation is given to medicines for diseases that have a prevalence of fewer than five in 10,000 individuals, are life-threatening or debilitating and have no adequate means of diagnosis or treatment. By receiving this designation, LMI has access to several benefits including fee reductions and special protections once florbetaben (18F) reaches the market.
A multi-center clinical trial is currently being conducted to evaluate the efficacy of florbetaben (18F) in diagnosing patients with different types of cardiac amyloidosis, including ATTR-CM.
In previous clinical trials evaluating the safety of florbetaben (18F), the most common adverse reactions were redness, pain and irritation at the injection site.
LMI is a pharmaceutical company that develops novel tracers to be used in PET scans, with a focus on neurological and cardiovascular imaging. Their mission is to promote the early diagnosis of a variety of chronic and life-threatening diseases.
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