Dr. Christian Schneider
Senior Medical Officer at the Danish Health and Medicines Authority
Titel: "Lessons learnt from the Marketing Authorisation of the first gene therapy authorised in the Western hemisphere"
Zeit: 14.00 Uhr
Ort: TWINCORE Lecture Hall
Kontakt: Prof. Dr. Ulrich Kalinke
Zusammenfassung: In July 2012, the Committee for Advanced Therapies (CAT) at the European Medicines Agency (EMA) recommended Marketing Authorisation for alipogene tiparvovec, the first gene therapy medicinal product to be authorised in the Western hemisphere. Its approval process was extensively discussed in the scientific community, sometimes critically. During the process, the opinions of the Committee for Medicinal Products for Human Use (CHMP) and the CAT differed, but both committees finally recommended approval of the medicine. This presentation provides insight into why the procedure was challenging, and takes the approval process for this medicinal product as a case study for regulation of orphan drugs and gene therapies in Europe.