European Shortages Monitoring Platform (ESMP): Implementation guide for national competent authorities
Quality control of medicinal product data submitted as per the legal requirement introduced by Article 57(2) of Regulation (EC) No. 726/2004
CHMP-CAT - D80-210 Overview to final EPAR - Rev 06.25 Revamp
Day 80 assessment report - Non-clinical template with guidance - Rev 06.25 Revamp
Agenda - Management Board meeting: 11-12 June 2025
Agenda of the COMP meeting 10-12 June 2025
Agenda of the CVMP meeting 10-12 June 2025
Orphan designation: genetically modified replication-incompetent herpes simplex virus-1 expressing collagen VII (beremagene geperpavec) Treatment of epidermolysis bullosa, 16/04/2018 Positive
Orphan designation: Adeno-associated virus serotype rh79 containing the human OTC gene, adeno-associated virus serotype rh79 encoding a meganuclease for targeted editing of the human PCSK9 gene Treatment of ornithine transcarbamylase deficiency, 09/12/2022 Positive
Orphan designation: Humanised IGg1 monoclonal antibody targeting human transferrin receptor conjugated to human iduronate-2-sulfatase Treatment of mucopolysaccharidosis type II (Hunter's syndrome), 26/02/2019 Positive