Committee of the Medical Department for Medicinal Products for Human Use recommended the first tool for gene therapy to the approval of relevant departments.
The recommendation must be approved by the European Commission, but the latter does not usually accept the decisions of this body.
Glybera intended patients (one or two people per million), which can not produce enough of lipoprotein lipase, which is crucial for fat digestion. With the help of the virus preparation sends man working copy of the gene involved in the development of this enzyme.
The first country to approve gene therapy, was China, it happened in 2004. Then it came to the treatment of cancer. Europe and the United States still does not follow suit, fearing above all the carcinogenic gene therapy.
Glybera, developed by the Dutch company uniQure, previously received negative reviews and the Committee for Medicinal Products for Human and the Committee on best practices for treatment of the same European medical management. Nevertheless, after a second more narrow trials in patients with severe cases of pancreatitis as a result of deficiency of lipoprotein lipase second issued a positive opinion in June, and now it was the turn of the first and the committee. Officials agreed on the fact that the advantages in this particular group outweigh the risks.
Ultimately recommended to use the drug only in exceptional circumstances. This allows you to approve a vehicle without the need for large-scale clinical trials, which, strictly speaking, it is impossible to carry out, when it comes to rare diseases. Total Glybera 27 patients received during the three tests. All further cases taking the drug will also be monitored for the accumulation of statistics.
The next step nameless until tool aimed at adenosine deaminase deficiency - Severe combined immunodeficiency. In this case, gene therapy is conducted ex vivo - on samples of bone marrow extracted from the body of the sick person, and then the patient is appropriate injection.
In drug development including pharmaceutical giant GlaxoSmithKline, which, experts believe, increases the chances of its approval.