On 20 July, the European Medicines Agency (EMA) published a press release that they recommend the first gene therapy (Glybera) for approval. Glybera intended to treat lipoprotein lipase (LPL) deficiency in patients with severe or multiple pancreatitis attacks, despite dietary fat restrictions.
Glybera is the first gene-therapy medicine to be recommended for authorisation in the European Union. Gene therapy medicines have the potential to cure genetic disorders by replacing a defective gene with a working copy, thus helping the body to recover functionality. Glybera uses an adeno-associated virus vector as the delivery vehicle to add working copies of the LPL gene into muscle cells to enable production of the enzyme in the cells.
The marketing-authorisation application for Glybera was submitted to the Agency in December 2009. In June 2011, both the CHMP and the Committee for Advanced Therapies (CAT) adopted negative opinions concerning the use of Glybera in the treatment of patients with LPL deficiency. During the re-examination, the CAT considered that there was scope for approval of Glybera with additional post-marketing studies, but the CHMP maintained its negative opinion in October 2011.
The CHMP recommended the granting of the marketing authorisation under ‘exceptional circumstances’. The company that markets Glybera will be required to provide data from a registry set up to monitor outcomes in patients treated with Glybera, which the Agency will review as they become available.
More details can be found in the EMA's press release "European Medicines Agency recommends first gene therapy for approval".
Axel H. Schroeder
CONCEPT HEIDELBERG (a service provider entrusted by the ECA Foundation)