Gene and Cell Therapy in Germany and the EU

Abstract

Gene and somatic cell therapy medicinal products as well as tissue engineered products are summarized under the term advanced therapy medicinal products in the EU. The Paul-Ehrlich-Institut has established a specialized division with various sections providing scientific advice and clinical trial authorization to applicants, thereby protecting patients’ health and supporting product development. Gene therapy medicinal products include products suitable for in vivo diagnostics, for the prevention of infectious diseases (such as vaccines) and the treatment of common diseases such as cancer, cardiovascular and infectious diseases, and of rare or orphan diseases such as inherited monogeneic diseases. Clinical trial authorization in Germany is provided by the Paul-Ehrlich-Institut and, if genetically modified organism-containing medicinal products are used, includes deliberate release authorization under consultation with the Federal Office for Consumer Protection and Food Safety (BVL). Firstmarketing authorization applications of gene therapy medicinal products developed for the treatment of cancer have been submitted to the European Medicines Agency (EMEA) and have been or are being reviewed by experts from national medicines agencies. Beneficial gene therapy treatment in clinical trials has been reported for infants suffering from adenosine deaminase deficiency-based severe combined immunodeficiency disease. Human somatic cell therapy medicinal products are mainly being developed for cancer immunotherapy; tissue engineered products contain viable cells able to replace, repair or substitute for human tissues or cells, e.g., after myocardial infarction. Chondrocytes are being used clinically for cartilage repair and an application for EU-wide marketing has been submitted to the EMEA. The main issues for cell-containing medicinal products are cell selection, differentiation, manipulation to obtain specific characteristics and avoiding pre-neoplastic alterations. In summary, special expertise is needed to develop advanced therapy medicinal products and has to be met by scientists specialized in regulatory aspects to support patient safety and product development. Consequently, the EMEA will establish the Committee for Advanced Therapies to review future marketing authorization applications.