European Medicines Agency gives first approval of gene therapy for rare disease

LONDON – The European Medicines Agency is recommending the first-ever approval of a gene therapy treatment in the West.

The EMA said in a statement Friday that Glybera, made by Dutch company UniQure, should be approved as a treatment for a rare inherited disease that makes people unable to digest fat. The treatment consists of a gene that makes a protein to break down fat.

Gene therapy is an experimental technique that holds promise to cure diseases by replacing genes that don’t work. It has never been approved in the EU or U.S.

The EMA previously rejected Glybera but reconsidered at the request of the European Commission. The agency is recommending approval under tough restriction.

Recommendations by the EMA are usually given final approval by the Commission.