Gene therapy move gives hope to patients with rare fat disorder
The European Medicines Agency (EMA) is recommending the first 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.
Last night, 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 US 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.
The EMA rejected Glybera three times but reconsidered at the request of the European Commission. The agency recommended approval under tough restrictions and will require the company to closely track patients.
"It is only meant for patients with the greatest need," said Monika Benstetter, an EMA spokeswoman, explaining the gene therapy was intended for people with no other treatment options.
Patients with lipoprotein lipase deficiency, the disease Glybera is intended to treat, often cannot eat a normal meal because it can lead to an extremely painful inflammation of the pancreas.
Jorn Aldag, CEO of uniQure, said the firm was developing similar treatments for other diseases, including Parkinson's. "We believe that like antibodies, gene therapy will one day be a mainstay in clinical practice," he said.