European Medicines Agency (EMA) has recommended European Union market approval for a gene therapy, Glybera, to treat patients with severe cases of a rare genetic condition called
lipoprotein lipase deficiency (LPLD). If the
treatment is approved by the European Commission, which usually follows the
recommendations of the EMA, Glybera will be the first commercially available
gene therapy in Europe and the USA.
is a watershed moment. Gene therapy holds incredible promise for people,
especially those with rare disease. It paves the way for the approval of other
treatments of this kind', said Dr Tim Coté, former director of the US Food and
Drug Administration's office of orphan products development.
to mutations in the LPL gene, patients with LPLD lack the necessary enzyme to break down fat from digested food,
which can cause fat globules to build up in the blood resulting in severe
abdominal pain and pancreatitis. The only way to manage the disease at present
is for patients to control the fat content in their diet. Glybera works by
using a virus vector to introduce a working copy of the gene.
Aldag, chief executive at UniQure, the company that is developing the drug,
said: 'Now, for the first time, a treatment exists for these patients that
not only reduces this risk, but also has a multi-year beneficial effect after
just one treatment'.
approval of Glybera has not been without problems. LPLD is very rare affecting one or two people per million, so it is difficult to assess the risks and
safety of the drug in the usual way. Glybera originally received a negative
response from the EMA, and the recent recommendation is only for use in
patients with a severe form of the disease.
therapy is aimed at patients with genetic diseases and essentially replaces
their faulty genes with working ones. The only other commercially available gene
therapy is Gendicine, which was approved for the Chinese market in 2004 to treat
therapy has received bad press in the past after the trial of a drug to treat
children with severe combined immunodeficiency was suspended due to some
participants developing leukaemia. Another setback to the development of gene
therapies was the death of a participant after he reacted adversely to the
injected virus in a gene therapy trial.