The European Medicines Agency is recommending the first-ever approval of a gene therapy treatment in the EU, in a significant move for a type of treatment that has so far failed to deliver on its promise to cure diseases.
In a statement on Friday, the EMA said Glybera, made by Dutch company uniQure, should be approved across Europe for the treatment of an extremely rare disorder that leaves people unable to digest fat. The treatment consists of a gene that makes a protein to break down fat.
Gene therapy is an experimental technique that tries to cure diseases by replacing genes that don't work. It has never been approved in the U.S. and most trials over the past two decades have failed. China was the first country to approve a gene therapy treatment in 2003 for cancer.
Scientists have struggled to find ways to deliver the genes safely, often by using a harmless virus. There are also concerns that inserting a gene at the wrong spot could cause cancer or that the body's immune system might attack the new gene and the virus used to deliver it.
The EMA previously rejected Glybera three times but it was reconsidered at the request of the European Commission. The agency recommended approval under tough restrictions and will require the company to set up a registry to closely track patients. Previous trials of the treatment only tested it in 27 patients.
