First Gene Therapy Approved by the European Commission - ECLT - Università di Pavia

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First Gene Therapy Approved by the European Commission

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First Gene Therapy Approved by the European Commission

The European Commission (EC) has granted Marketing Authorization for Glybera, a gene therapy developed by uniQure which treats lipoprotein lipase deficiency (LPLD).

Patients who suffer from LPLD are unable to metabolize the fat particles carried in their blood and this leads to inflammation of the pancreas. LPLD is a rare and genetically inherited disease.

The only way in which patients have so far been able to manage the disease is to follow a very low fat diet. Glybera is the first medical treatment option given to patients of LPLD. The therapy uses a virus to infect muscle cells with a gene variant of  lipoprotein lipase (LPL), the  LPLS447X  

The Authorization is valid in all 27 Member States of the European Union. uniQure is also preparing to apply for regulatory approval in the U.S., Canada and other markets.

Jorn Aldag, the CEO of  uniQure stated: “The final approval of Glybera from the EC marks a major step forward in making gene therapies available not only for LPLD but also for a large number of rare diseases with a very high unmet medical need”.

For more information on this issue please refer to the following websites:

http://www.uniqure.com/news/167/182/uniQure-s-Glybera-First-Gene-Therapy-Approved-by-European-Commission.html


http://ec.europa.eu/health/documents/community-register/html/h791.htm


http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002145/smops/Positive/human_smop_000235.jsp&mid=WC0b01ac058001d127





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