ATMPs in Germany - Glybera®

Glybera^® (alipogene tiparvovec)

Glybera (active substance: alipogene tiparvovec) was approved for treatment of familial lipoprotein lipase deficiency (LPLD) in October 2012. It was the first approved gene therapy for enzyme deficiency. The market authorization was withdrawn and expired in October 2017. Consequently, Glybera is no longer available in Germany.

🏆German HTA rating:

The German HTA rating in May 2015 resulted in a hint for a non-quantifiable additional benefit since the scientific data did not allow quantification. As three non-randomized, non-controlled studies (and no RCT) were submitted for HTA, the additional benefit was granted by law, because of the orphan drug status of Glybera.

💵 Launch price: € 43,870.00 per vial. Depending on patient’s weight up to 30 vials of Glybera might be required per treatment. Glybera is used as a one-time treatment. Reimbursement price: not listed (no official negotiated reimbursement price was listed, as Glybera is only used in the in-patient setting)

📘 Special features of reimbursement:

As Glybera was only used in a few highly specialized centers and due to the extreme rarity of the disease LPLD, reimbursement of Glybera was subject to a case-by-case decision by the respective health insurance company.

💡 Glybera was the second ATMP assessed in German HTA. In the orphan indication LPLD with an expected patient numbers of 17 – 35 per year in Germany, the German HTA body also demanded an RCT, even though it is hardly possible to realize. Only the orphan drug status of Glybera allowed for granting of an additional benefit.