Gene therapy to be authorised for first time in EUBMJ 2012; 345 doi: https://doi.org/10.1136/bmj.e5009 (Published 23 July 2012) Cite this as: BMJ 2012;345:e5009
- Geoff Watts
The European Medicines Agency has recommended that alipogene tiparvovec (marketed as Glybera) be authorised for sale in the European Union. Intended to treat lipoprotein lipase deficiency in patients with severe pancreatitis, it is the first gene therapy in Europe to be recommended for this status and is yet to be approved in the United States.
The deficiency is a genetic disorder thought to affect no more than one or two people in a million. The new treatment relies on an adeno-associated virus to deliver functional copies of the lipoprotein lipase gene to the body’s muscle cells.
“It’s fantastic news …
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