Alipogene tiparvovec
| Gene therapy | |
|---|---|
| Target gene | LPL |
| Vector | Adeno-associated virus 1 |
| Clinical data | |
| Trade names | Glybera |
| Routes of administration | Intramuscular injection |
| ATC code | |
| Legal status | |
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| Identifiers | |
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Alipogene tiparvovec, sold under the brand name Glybera, is a gene therapy treatment designed to reverse lipoprotein lipase deficiency (LPLD), a rare recessive disorder, due to mutations in LPL, which can cause severe pancreatitis.[1] It was recommended for approval by the European Medicines Agency in July 2012, and approved by the European Commission in November of the same year. It was the first marketing authorisation for a gene therapy treatment in either the European Union or the United States.[2][3]
The medication is administered via a series of injections into the leg muscles.[4]
Glybera gained infamy as the "million-dollar drug" and proved commercially unsuccessful for a number of reasons.[4][5] Its cost to patients and payers, together with the rarity of LPLD, high maintenance costs to its manufacturer uniQure, and failure to achieve approval in the US, led to uniQure withdrawing the drug after two years on the EU market. As of 2018, only 31 people worldwide have ever been administered Glybera, and uniQure has no plans to sell the drug in the US or Canada.[4][5]
- ^ Pollack A (20 July 2012). "European Agency Backs Approval of a Gene Therapy". The New York Times.
- ^ Gallagher J (2 November 2012). "Gene therapy: Glybera approved by European Commission". BBC News. Retrieved 15 December 2012.
- ^ Richards S (5 November 2012). "Gene Therapy Arrives in Europe". The Scientist. Retrieved 16 November 2012.
- ^ a b c Cite error: The named reference
cbc-glyberawas invoked but never defined (see the help page). - ^ a b Warner E (20 April 2017). "Goodbye Glybera! The World's First Gene Therapy will be Withdrawn". Labiotech. Retrieved 23 June 2019.