Market Overview

AlphaCore Granted Orphan Drug Designation for ACP-501 (rhLCAT) by European Medicines Agency


AlphaCore Pharma, a biopharmaceutical company developing ACP-501 (rhLCAT) for the treatment of high risk atherosclerosis and serious lipid metabolism disorders, today announced it has received orphan drug designation from the European Medicines Agency (EMA) for ACP-501 for the treatment of familial LCAT deficiency (FLD). This EMA designation follows an earlier FLD orphan drug designation granted by the US Food and Drug Administration.

“The EMA's grant of orphan drug designation to ACP-501 for the treatment of FLD represents an important milestone in the development of this program, and it highlights the significant unmet medical need. With this announcement, we are another step closer towards bringing enzyme replacement therapy to these patients who currently have no other treatment options available,” commented Bruce Auerbach, President of AlphaCore Pharma.

Familial LCAT deficiency is a rare, inherited condition that causes lipid accumulation in the eyes, red blood cells and kidneys. Symptoms of the disease include corneal opacity (clouding of the cornea), hemolytic anemia and kidney disease which can lead to kidney failure. Treatment with ACP-501 replaces the missing enzyme in FLD patients with a goal of restoring normal kidney function.

About Orphan Drug Designation

EMA's Orphan Medicinal Product Designation is designed to promote the development of drugs that may provide significant benefit to patients suffering from rare, life-threatening diseases, i.e., those affecting fewer than 250,000 people in the European Union (EU). This designation will provide ten years of marketing exclusivity if the product candidate is approved for marketing for the designated orphan indication in the EU. It also provides special incentives for sponsors, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees during development or at the time of application for marketing approval.

About AlphaCore Pharma

AlphaCore Pharma is a privately held Ann Arbor-based biotechnology company formed in 2007 to develop recombinant human lecithin cholesterol acyltransferase (rhLCAT) as a novel therapeutic agent for the treatment of high risk atherosclerosis and serious lipid metabolism disorders. ACP-501 was evaluated in a recent Phase 1 study that demonstrated safety, tolerability and desirable lipid effects in patients with coronary artery disease. Additional information is available at

AlphaCore Pharma
William Brinkerhoff, 734-619-8801

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