Spark's Luxturna becomes US's first approved gene therapy

Children’s Hospital of Philadelphia today joins with other research leaders in celebrating the U.S. Food and Drug Administration’s approval of Spark Therapeutics’ innovative gene therapy for the treatment of a rare inherited form

The FDA has approved a landmark gene therapy for vision loss-the first ever to fix a faulty gene within a patient's body-but the therapy's owner isn't yet saying how much the drug will cost.

The therapy is a recombinant adeno-associated viral (AAV) vector serotype 2, expressing the gene for human retinal pigment epithelial 65 kilodalton protein (AAV2-hRPE65v2) which was found to be 93% effective in improving vision in patients with inherited retinal dystrophy (IRD) in Phase III trials. The approval is a landmark moment for a field riddled with ups and downs since the first gene therapy trial began almost three decades ago.

Luxturna becomes the first directly-administered gene therapy to get a green light in the USA in a year that has also seen the first approval by the U.S. regulator of two CAR-T therapies for blood cancers which involve genetically modifying cells outside the body.

"The buildup for gene therapy has been without numerous triumphs and really a couple of disappointments, so chalk this one up in the win section", said Valle, a geneticist and pediatrician at Johns Hopkins University, who was not engaged with Luxturna's advancement.

"I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable diseases", Dr. Scott Gottlieb, commissioner of the FDA, said in a statement announcing the approval.

The approval is a culmination of more than 25 years of studies on congenital blindness by married-couple team Bennett and Maguire at Penn and CHOP, starting with pioneering work in mice and dogs.

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This is the third gene therapy approved by FDA since August 2017. It will release new guidance for gene therapy developers next year. In 2000, she tested a gene therapy for genetic retinal disease in dogs. He applauded researchers at the University of Pennsylvania for decades of work that led to the treatment. The first gene therapy product costs $475,000 per patient. Light causes an eye pigment to change from a scrunched-up form called cis-retinal to an extended form called trans-retinal.

To begin the cycle anew, the RPE65 enzyme reverts trans-retinal back into cis-retinal. This loss of vision, often during childhood or adolescence, ultimately progresses to complete blindness. The normal genes are delivered by a modified adenovirus, similar to the ones responsible for the common respiratory infections.

Although many mutations in RPE65 can lead to blindness, in theory Luxturna should be able to help them all.

RPE65-mediated IRD affects somewhere between 1,000 and 2,000 people in the USA and a similar number in the European Union and occurs when both parents pass on the RPE65 mutation to a child.

Goldman Sachs expert Salveen Richter predicts Luxturna will cost $500,000 per infusion, or $1 million for the two eyes. Within the liquid is a gene therapy virus that contains a healthy version of the gene.

One year after treatment, patients who got the infusion demonstrated critical enhancements in exploring the impediment course at low light levels contrasted with the individuals who did not get the therapy. Meant to encourage development of therapies for rare pediatric diseases, the transferable vouchers have been sold for up to $250 million between drug companies.

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