ABIONYX Pharma Receives Positive Opinion From EMA Within the Framework of the Orphan Drug Designation Process for CER-001 for the Rare Disease LCAT Deficiency

ABIONYX Pharma (FR0012616852 – ABNX – PEA PME eligible), a next-generation biotech company dedicated to the discovery and development of innovative therapies, announced today that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has issued a positive opinion on the company’s application for Orphan Drug Designation (ODD) for its drug candidate CER-001, as a potential treatment for lecithin-cholesterol acyltransferase (LCAT) deficiency clinically characterized by hemolytic anemia and renal failure, most often leading to renal transplantation, and corneal opacities. CER-001 is a first-in-class bio-HDL mimetic that directly targets a key underlying metabolic defect of LCAT deficiency.

The culmination of a new strategy to use CER-001 in an ultra-rare renal disease

This positive opinion from the COMP of the EMA for an orphan drug designation for bio-HDL is of strategic importance for the development of our product. This designation offers an important advantage by providing access to the centralized marketing authorization procedure as well as 10 years of market exclusivity in the European Community from the date of obtaining a marketing authorization. This positive opinion is also a recognition of the need for innovative treatments for patients with LCAT deficiency, a serious and lifelong disease. Several nATU applications have been received necessitating the decision to secure biomanufacturing to produce clinical batches for patient use and a European regulatory filing. CER-001 is the only bio-HDL to receive this important designation in Europe and the first potential disease-modifying treatment for patients with LCAT deficiency.

Orphan drug designation in the European Union (EU) is granted by the European Commission on the basis of a positive opinion issued by the COMP of the EMA. To obtain this designation, a drug candidate must be intended to treat a severely debilitating or life-threatening condition that affects fewer than 5 in 10,000 people in the EU, and there must be sufficient clinical or non-clinical data to suggest that the investigational drug can produce clinically relevant results. Orphan drug designation by the EMA provides companies with certain benefits and incentives, including assistance with clinical protocols, differentiated assessment procedures for health technology assessments in certain countries, access to a centralized marketing authorization procedure valid in all EU member states, reduced regulatory fees, and 10 years of market exclusivity from the time a marketing authorization is granted.

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