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news.The European Medicines Agency (EMA) has granted orphan drug status to AlphaCore Pharma's ACP-501 (rhLCAT) to treat familial LCAT deficiency (FLD).
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FLD causes lipid deposition in eyes, red blood cells and kidneys which can lead to corneal opacity, hemolytic anemia and kidney failure respectively.
ACP-501, being developed to treat atherosclerosis and lipid metabolism disorders, is expected to replace the related enzyme to regain the lost kidney function in FLD patients.
AlphaCore president Bruce Auerbach said the ACP-501’s orphan drug designation obtained from EMA represents the significant unmet medical need of FLD.
"With this announcement, we are another step closer towards bringing enzyme replacement therapy to these patients who currently have no other treatment options available," Auerbach added.
The USFDA had previously issued FLD orphan drug designation to ACP-501.