(BFM Bourse) – The European Medicines Agency has voted in favor of the orphan drug status for CER-001 as a potential treatment for lecithin-cholesterol acyltransferase (LCAT) deficiency, an ultra-rare disease. Abionyx is counting on rapid approval, the molecule having already been administered by derogation to several patients.
Abionyx is in the starting blocks, ready to distribute its CER-001 therapy (still awaiting the attribution of a trade name) as soon as it is approved. The molecule is in the process of obtaining orphan drug status from the European authorities, and supply and production are secure. This important regulatory advance, with regard to the group’s repositioning strategy in renal and ophthalmological diseases after its failure a few years ago in the treatment of cholesterol, allows Abionyx to accelerate from 15.69% to 1 , 15 euro Wednesday afternoon, a high for a month.
The Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has issued a positive opinion on the application for designation of an orphan medicinal product submitted by Abiony for its drug candidate CER-001 as a potential treatment for impairment in lecithin-cholesterol acyltransferase. In practice, the opinion of the COMP is always followed, so that obtaining this status is only a matter of formalism.
As the biotech underlines, this marks a real achievement in the execution of its strategy in an ultra-rare renal disease, lecithin-cholesterol acyltransferase (LCAT), affecting less than one person in a million. This serious and permanent disease is characterized on the one hand by hemolytic anemia and renal failure, most of the time leading to transplantation, and on the other hand, by corneal opacities.
CER-001 is the world’s first bio-HDL (the “good” cholesterol) mimetic, which directly targets the metabolic defect underlying LCAT deficiency. This is the first potential treatment likely to modify the course of the disease, and it has already been administered in practice to patients within the framework of nominative Temporary Authorizations for Use, with the agreement of the authorities concerned, testifying to the interest of practitioners in the face of a therapeutic need that has hitherto been unsatisfied.
The orphan drug designation provides various advantages, including access to the centralized marketing authorization procedure as well as ten-year commercial exclusivity for the European Community.
As LCAT deficiency also affects the cornea, this first orphan designation paves the way for the development of Abionyx in ophthalmology, as the firm previously announced as part of the expansion of the innovation potential of CER- 001 and its pleiotropic action as a natural recombinant HDL.
In addition to ATUn, Abionyx Pharma is also conducting a phase 2a clinical study evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of treatment with CER-001 in adults with sepsis at high risk for acute kidney injury, a disease including prevalence is estimated at over 2 million people worldwide.
Guillaume Bayre – © 2021 BFM Bourse
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