By Naomi Kresge - 2012-07-20T19:16:23Z

Europe is on the cusp of approving the first gene therapy to be sold on the market in a western nation, a product from UniQure BV designed to treat a rare disease that disrupts fat production in the body.

The drug, called Glybera, won the backing of an advisory panel to the European Commission on its fourth attempt, the group said in a statement today. The Commission, which makes the final decision, usually follows the committees recommendation.

The move comes after 20 years of experimentation into a technology that in the past has been haunted by highly- publicized failures, including the death of 16-year-old Jessie Gelsinger in the U.S. in 1999. More recently, success in trials by Amsterdam-based UniQure and the U.S.-based companies Sangamo BioSciences Inc. (SGMO) and Neurologix Inc. (NRGXQ) are reviving the field.

This is good news for the field, definitely, said Savio Woo, a professor of genetics and genomic sciences at the Mount Sinai School of Medicine in New York. The technology has been really making a lot of progress, and a positive approval was more or less expected to come sometime.

UniQure began preparations a few weeks ago for meetings with the U.S. Food and Drug Administration to seek approval for Glybera, said Jorn Aldag, UniQures chief executive officer.

The treatment targets lipoprotein lipase deficiency, or LPLD, a rare fat-processing disorder that spurs severe or multiple pancreatitis attacks in about one or two people among every million in the population. The therapy is administered only once to be effective.

Glybera may have peak annual sales between 50 million euros ($61.1 million) and 300 million euros, depending on how its priced, Aldag said in an interview.

Its phenomenal because its the first gene therapy the EMA has approved, he said.

While two gene therapy products are sold in China, none are approved in Europe or the U.S., according to Mount Sinais Woo.

See the article here:
UniQure’s Glybera Wins EU Backing as First Gene Therapy

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