Abionyx Pharma: EMA positive notice for CER-001 in LCAT Deficiency

Abionyx Pharma, a new generation biotechnology company dedicated to the discovery and development of innovative therapies based on the only recombinant apolipoprotein apoA-1 in the world, submitted on July 11, 2024 a demand formed by scientific opinions in the Agence européenne des médicaments (EMA) for recombinant AI human apolipoprotein CER-001, in the treatment of lecithin-cholesterol acyltransferase (LCAT) deficiency. This demand completes the ongoing review process, with clinical data and partial chemistry, manufacturing and controls (CMC) data submitted by ABIONYX Pharma.

Abionyx Pharma at the request of the opinion of the Comité des médicaments à usage humain (CHMP), committee of the Agence européenne des médicaments responsible for the evaluation of médicaments à l’usage humain, to the proposal to submit, during the dépôt du dossier relatif au CER-001 en vue de l’octroi de l’approbation conditionalnelle de UE in l’indication de LCAT deficience, les données de deux prospective lots de validation du procédé relatif à la fabrication du biomedicament (which it includes the substance apoA- Jo called DS and the medicinal product called DP).

The EMA concludes that the proposal to submit the data from 2 prospective batches of validation of the procedure related to the manufacture of the biomedicine during the demand for marketing authorization could be admissible.

As recommended, Abionyx Pharma will pursue the viral safety development plan, method description and validation specificity until the submission of the MA.

Abionyx Pharma provided CER-001 in the framework of a nominative compassionate use to eight patients with LCAT deficiency in four European countries. All patients have completed six months of treatment. As agreed in the previous opinion of the CHMP, these cases constitute the clinical basis of the submission of the MA.

On the basis of the scientific opinion of the EMA, the Company has now clarified the conditions required to initiate the submission process of an AMM.

Source and display: ABIONYX Pharma