The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Glybera (alipogene tiparvovec) for marketing in the EU. Glybera is the first gene therapy medicine to be recommended for authorization in the European Union, and is produced by uniQure, an Amsterdam-based company focused on gene therapies.
Glybera is intended to treat a rare hereditary disorder, lipoprotein lipase (LPL) deficiency. LPL is an enzyme responsible for breaking down fats, and patients who do not produce enough of the enzyme have elevated blood fat levels that can result in severe pancreatitis. Glybera introduces a normal copy of the LPL gene into muscle cells using a delivery vector derived from adeno-associated virus.
“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 Tomas Salmonson, acting Chair of the CHMP. “Our established ways of assessing the benefits and risks of Glybera were challenged by the extreme rarity of the condition and also by uncertainties associated with data provided. In close cooperation with the CAT we have worked out a way to ensure robust and close follow-up of the quality, safety and efficacy of Glybera while giving patients who have to live with this rare disease access to a medical treatment.”
According to a press release from uniQure, as part of the approval, treatment with Glybera will be offered through dedicated centers of excellence with expertise in treating LPL deficiency and by specially trained doctors to ensure ongoing safety. CHMP has also stipulated that uniQure will be required to provide data from a registry set up to monitor outcomes in patients treated with Glybera.CHMP’s opinion on Glybera will be sent to the European Commission for the adoption of a marketing authorization. UniQure indicated that it is preparing to apply for approval in the US, Canada, and other markets.