On October 25, 2012, the European Commission approved alipogene tiparvovec for the treatment of adult patients with lipoprotein lipase deficiency (LPLD) and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions.18,19 The indication was restricted to patients with detectable levels of lipoprotein lipase (LPL) protein18. LPLD is a rare genetic disorder caused by deficiency of LPL, which can cause severe pancreatitis, despite dietary fat restrictions.20

Alipogene tiparvovec, an adeno-associated virus serotype 1 (AAV1)-based vector engineered to express LPL in the muscle tissue21, became the first AAV gene therapy product approved in Europe19

In October 2017, alipogene tiparvovec was withdrawn from the market due to extremely limited usage.22 The withdrawal was not related to any risk–benefit concerns.22 Reasons cited for the withdrawal include the high manufacturing costs, extensive maintenance activities associated with its marketing authorization, a reduction of only 50% in the development of pancreatic disease, and approval in a very small patient population.22,23