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 committee’s 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) (SGMO) and Neurologix Inc. (NRGXQ) (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, UniQure’s 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 it’s priced, Aldag said in an interview.
“It’s phenomenal because it’s 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 Sinai’s Woo.
Gene therapy is “not a cure-all for all diseases,” Woo said in a telephone interview. “But it certainly has its utility in the treatment of many debilitating diseases that will complement existing treatments.”
Glybera was developed by Amsterdam Molecular Therapeutics. (AMT) The company sold itself to UniQure this year after European authorities initially rejected the drug.
Genes contain instructions for cells to make proteins that carry out chemical reactions in the body, help the immune system fight infections and form the basis of skin, muscle and bone. When a gene is defective and can’t make the correct protein, disease can occur.
The idea of gene therapy is to treat disease by replacing malfunctioning genes with healthy ones.
Use of gene therapy has been controversial, and not always successful. Gelsinger died of a massive immune response within hours of being treated for a liver disease at the University of Pennsylvania in Philadelphia in 1999. That failure was followed in 2007 by the death of a woman who received a modified gene in an arthritis trial.
Sangamo BioSciences Inc., based in Richmond, California, is in the second of three phases of testing required on a gene therapy to tackle HIV, while Neurologix Inc., of Fort Lee, New Jersey, is studying use of the treatment against Parkinson’s Disease.
Bluebird Bio, a closely held company in Cambridge, Massachusetts, is developing therapies for blood conditions including beta thalassemia and sickle cell disease after clinical trials reported positive results. Bluebird is backed by Sanofi’s Genzyme Corp., also in Cambridge, and by venture capital firms.
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