Alipogene Tiparvovec-A Gene Therapy For Lipoprotein Lipase Deficiency

Alipogene Tiparvovec is a gene therapy developed for the treatment of Lipoprotein Lipase Deficiency (LPLD), very rare inherited condition that is coupled with increased levels of fat in the blood which can cause severe pancreatitis. It is a first therapeutic cure available for LPLD patients. Earlier, patients were predominantly treated with severe dietary fat restriction and the use of medium-chain triglycerides with persisting risk of disease progression or pancreatitis. Alipogene Tiparvovec is marketed under the trade name Glybera.

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Alipogene tiparvovec was developed by Amsterdam Molecular Therapeutics (AMT), which was acquired by uniQure in April 2012.In October 2012, uniQure received marketing authorization approval for Glybera from the European Commission (EC) for treating patients with LPLD who have chronic and acute pancreatitis attacks. Alipogene tiparvovec is the first gene therapy to be licensed in Europe and is administered intramuscularly into the leg muscle. Glybera is a significantly expensive treatment costing approximately USD 1.6 million.

Lipoprotein lipase deficiency prevalence is estimated at one to two per million. It is the most ordinary cause of hyperchylomicronaemia, a situation in which triglycerides are not offloaded from chylomicrons because of dysfunctional LPL, causing continuous and extremely high levels of plasma chylomicrons and severe hypertriglyceridaemia. Alipogene Tiparvovec has been tested in three interventional clinical studies conducted in Netherlands and in Canada, in which a total of 27 LPLD patients participated. In all three clinical trials, Glybera was well tolerated, with no relevant safety issues observed. Data from these clinical trials show that a single dose administration of Glybera resulted in a long-term biological activity of the LPL protein and reduction in the episodes of acute pancreatitis. Interventional studies have always shown that alipogene tiparvovec is well tolerated without dose-limiting toxicity.

The most common side effect reported with Glybera was pain in the legs, tiredness, headache, and hyperthermia (high body temperature). Glybera must not be used in patients with increased risk of bleeding and muscle disease, immunodeficiency (weakened immunity), along with anti-coagulant medicines, which can increase bleeding. Patients must also not use oral contraceptives along with Glybera.

There are certain government organizations which provide funding for research and development of alipogene tiparvovec which acts as a driving factor for global gene therapy market. The UCLA Center for Pancreatic Diseases (CPD) is one of them. The clinical arm of the UCLA Center for Pancreatic Diseases applies a multidisciplinary approach to the evaluation and treatment of patients with pancreatic diseases. However, the high cost associated with research activities and sunk costs involved in research & development activities are restraining the immune checkpoint inhibitors market.

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Geographically, the global alipogene tiparvovec market is segmented into North America, Europe, Asia-Pacific (APAC) and Rest of the World. The European Medicines Agency (EMA) has approved Glybera, for sale across Europe. Glybera is the first gene therapy to be approved in Europe and North America and offers an effective therapeutic treatment for people with LPLD. LPLD is more prevalent in Canada and South Africa according to a published study by Elsevier Ireland Ltd. in 2010. Thus major market for alipogene tiparvovec lies in the Canada and African region where the prevalence of LPLD is high.

The marketing authorization holder of alipogene tiparvovec drug is UniQure biopharma B.V. Other major market players involved in the research and development of alipogene tiparvovec drug are, International Antiviral Therapy Evaluation Center, the Clinical Trial Company Limited and ICON.

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