ABIONYX Pharma Gets EMA Green Light for CER-001 in Rare LCAT Deficiency

ABIONYX Pharma (FR0012616852 - ABNX - PEA PME eligible), a cutting-edge biotech company focused on developing innovative therapies, has completed a key step in advancing CER-001, the world’s only natural recombinant apoA-I, for lecithin-cholesterol acyltransferase (LCAT) deficiency treatment. On July 11, 2024, the company formally submitted a request for scientific advice to the European Medicines Agency (EMA), marking the completion of its rolling review process. This included clinical data and partial Chemistry, Manufacturing, and Controls (CMC) information.

ABIONYX Pharma sought the EMA’s Committee for Medicinal Products for Human Use (CHMP) feedback on whether submitting data from two prospective process validation batches for CER-001’s Drug Substance and Drug Product at the time of filing for conditional EU approval would be acceptable. The EMA has agreed that this approach could be feasible.

Moving forward, ABIONYX Pharma will continue refining its viral safety protocols, method descriptions, and validation processes in preparation for submitting its Marketing Authorization Application (MAA). Meanwhile, CER-001 has been provided on a compassionate use basis to eight patients with LCAT deficiency across four European countries, all of whom have now completed six months of treatment. These patient outcomes will be pivotal in the clinical evidence presented in the MAA submission. Following the EMA's advice, ABIONYX Pharma is now ready to proceed with its MAA submission plan.