ABIONYX strengthens its commitment against LCAT Deficiency

On the occasion of World Rare Disease Day, ABIONYX Pharma, a company specializing in the development of biomedicines, announces its progress in the fight against LCAT Deficiency, also known as Norum Disease. This commitment includes the creation of a patient association and assistance with early diagnosis. The company is also working on preparing a Marketing Authorization Application (MAA) after the exemption of the clinical phase 3, validated by the EMA.

LCAT Deficiency, affecting less than one in a million people, causes severe renal and ophthalmologic complications. ABIONYX is developing a biomedicine for these rare diseases, based on orphan disease designations obtained in Europe and the United States. These recognitions will accelerate the marketing authorization process and provide extended market exclusivity. This progress marks a critical step towards providing an innovative treatment for affected patients.