New gene-therapy may be approved in EU

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Follow @WDMEllaCopeland New treatment Glybera has been recommended for marketing in the European Union as a treatment for severe and multiple pancreati...

New treatment Glybera has been recommended for marketing in the European Union as a treatment for severe and multiple pancreatitis attacks due to lipoprotein lipase deficiency (LPL).

Glybera (alipogene tiparvovec) is the first gene-therapy medicine to be recommended for authorisation in the European Union. Designed to replace defective genes with a working copy, the gene-therapy treatment Glybera will add working copies of the LPL gene into muscle cells to enable production of the enzyme in the cells.

LPL deficiency is an  extremely rare inherited disorder estimated to affect one or two people per million. Due to a defective gene, patients with this disorder cannot produce enough LPL, an enzyme responsible for breaking down fats. So far, management of patients with the disorder consists of strict reduction of dietary fat to less than 20% of the daily caloric intake. It is very difficult to comply with such a dietary regimen and as a consequence many patients experience life-threatening pancreatitis attacks requiring admission to hospital.

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended the granting of market authorisation in ‘exceptional circumstances’, based on the requirement that the company marketing Glybera will be required to provide data from a registry set up to monitor outcomes in patients treated with Glybera.

 “The evaluation of this application has been a very complex process, but the use of Glybera in a more restricted indication than initially applied for, which targets the patient population with greatest need for treatment, and additional analyses by the Committee for Advanced Therapies (CAT) have added to the robustness of the data provided and allowed the CHMP to conclude that the benefits of Glybera are greater than its known risks”, said Dr Tomas Salmonson, acting Chair of the CHMP.

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