FDA CRL - Recordati Rare Diseases Inc

Recordati Rare Diseases Inc. received a Complete Response Letter (CRL) from the FDA for its New Drug Application (NDA 211302) for Cystadrops (cysteamine ophthalmic solution) 0.37%. The FDA cited significant issues preventing approval under section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act. A primary concern is that the manufacturing facility, Baccinex SA, was found non-compliant with current Good Manufacturing Practice (CGMP) regulations (21 CFR 210 and 211) during a recent inspection. The FDA requires satisfactory resolution of these deficiencies and assurance that all facilities involved comply with CGMP. Further, the FDA identified inadequacies in the methods, facilities, and controls for manufacturing, processing, packing, and holding the drug product, impacting its identity, strength, quality, purity, stability, and bioavailability. Specific unresolved issues include insufficient data for key manufacturing parameters, the need for clarification on certain processes, and required revisions to the master batch record. The company must also provide results from a simulated manufacturing study. To address these concerns, Recordati must resolve all CGMP deficiencies, submit comprehensive data supporting manufacturing parameters, update and revise manufacturing records, and provide the results of the simulated study. Additionally, an updated safety summary, a stability update including particulate matter testing, and a complete analytical method transfer report are required. The proposed labeling also needs revision according to current FDA guidelines. A full resubmission addressing all deficiencies is required within one year.