On 19 July 2012, after a second evaluation which involved the adoption of a formal opinion of the Agency’s Committee for Advanced Therapies (CAT), the Committee for Medicinal Products for Human Use (CHMP) concluded that Glybera’s benefits outweigh its risks and recommended the granting of a marketing authorisation.
Glybera is a gene-therapy medicinal product constructed with an adeno-associated viral vector carrying the gene for lipoprotein lipase protein, which is expressed in muscle after administration. Adenoviruses usually infect mucosal membranes of the respiratory and reproductive tracts, but here, an adenovirus was modified to introduce a copy of the LPL gene, into patients that do not express the protein. This virus cannot replicate itself, and is not transmissible between people.
The medicine will be used to treat adult patients diagnosed with familial lipoprotein lipase deficiency (LPLD) and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions. The diagnosis of LPLD has to be confirmed by genetic testing. The indication is restricted to patients with detectable levels of LPL protein (see opinion). The disease is inherited, may effect one to two people per million.
The manufacturer, UniQure, will next apply for approval in the US and Canada.