uniQure, has decided not to renew the Marketing Authorization of Glybera in the EU. This decision is not related to any safety, efficacy or quality issue

The aim of the study is to provide further confirmatory evidence of clinical benefit in LPLD patients treated with alipogene tiparvovec by assessing both the "clinical response" (as defined by a range of parameters), and "the metabolic response" (postprandial CM metabolism) in LPLD patients with and without an immunosuppressant regimen.

This is a prospective, interventional, randomised, open-label, parallel group study evaluating the clinical response as well as the dynamics of postprandial chylomicron metabolism in patients treated with alipogene tiparvovec with and without immunosuppressants. The study will be conducted in 12 LPLD patients who will be randomised into the Immuno+ (cyclosporin and mycophenolate mofetil) or the Immuno- group.

Main inclusion criteria are:

• Patients with a history of severe or multiple pancreatitis attacks despite dietary fat restriction.
• Genetically confirmed diagnosis of LPLD
• Post-heparin plasma LPL protein mass > 5% of normal
• LPL activity ≤20% of normal (in post- heparin plasma)
• Fasting plasma TG concentration >10 mmol/L.

Main exclusion criteria are:

• Females with a positive pregnancy test or who are breastfeeding, or on contraceptive use.
• Patients with a positive HIV, Hepatitis B, Hepatitis C or being positive for tuberculosis.
• Patients under treatment with antiplatelet or other anti-coagulants.
• Patient allergic to or having a condition that prohibits the use of immunosuppressants.