The Need for Increased Clarity and Transparency in the Regulatory Pathway for Gene Medicines in the European Union

At the October 2011 meeting of the European Society of Gene and Cell Therapy (ESGCT) in Brighton, UK, the society hosted an excellent symposium in collaboration with the Committee on Advanced Therapies (CAT) of the European Medicines Agency (EMA), with a view to discussing problems and road blocks that biotech companies face when trying to bring Advanced Therapy Medicinal Products (ATMP) through the regulatory process. Overshadowing the symposium was the recent decision by the EMA to reject approval of Amsterdam Molecular Therapeutic's Glybera (alipogene tiparvovec) despite an approval recommendation by the CAT.