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news.Abionyx Pharma has received orphan-drug designation (ODD) from the US Food and Drug Administration (FDA) to the Bio-HDL CER-001 to treat lecithin-cholesterol acyltransferase (LCAT) deficiency.
CER-001 has been approved to treat LCAT deficiency presenting as kidney dysfunction and/or ophthalmologic disease. Credit: Robina Weermeijer on Unsplash.
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The first-in-class bio-HDL mimetic CER-001 works by directly targeting a key underlying metabolic defect of LCAT deficiency.
Its anti-inflammatory properties and/or the increase in the Reverse Cholesterol Transport (RCT) can help in preventing the kidney function decline and improve vision in LCAT patients.
CER-001 is claimed to be one of the most advanced biomedicines and a potential new treatment for kidney diseases, sepsis or Covid-19, as well as ophthalmologic diseases that include lipid abnormalities.
The FDA granted the ODD status for the drug to treat partial LCAT deficiency, called as Fisheye Disease, as well as complete LCAT deficiency present with renal symptoms and corneal opacities.
In July last year, CER-001 received ODD status from the European Medicines Administration (EMA) to treat LCAT deficiency.
Abionyx Pharma CEO Cyrille TUPIN said: “We are pleased to have received ODD for CER-001 just eight months after the ODD in Europe and one week after the first positive clinical results in Covid-19.
“These OD designations from the FDA underscore the importance of bringing this important therapeutic option to patients with LCAT Deficiency both as a kidney disease and as an ophthalmic disease.
“We look forward to presenting new clinical results in the coming months and providing additional insight into the potential of our Bio-HDL therapy platform in both kidney diseases and ophthalmologic diseases.”
The company noted that CER-001’s positive clinical results in LCAT disease have been published previously.
