This is the first time the FDA has approved a gene therapy that treats an inherited disease.
The Food and Drug Administration announced on Tuesday that it has approved a gene therapy that targets a rare form of childhood blindness. This is the first time the agency has ever given approval for a gene therapy that treats a hereditary disease.
The FDA will allow the start-up, Spark Therapeutics, to release its new gene therapy called Luxturna to the market. While a spokeswoman representing the pharmaceutical company has stated that they won’t disclose the price tag until January, Wall Street analysts believe the therapy will be at least $1 million.
Spark CEO, Jeffrey Marrazzo, said the FDA announcement was a watershed moment in opening the field of gene therapy to regular consumers. The new treatment may, in turn, allow the development of gene therapies for other conditions that currently do not have notable breakthroughs, according to Marrazzo.
Luxturna is no different than any other gene therapies, in the sense that it inserts a functional piece of DNA into cells in order to fix a defective gene. Spark’s new therapy targets a disease known as retinal dystrophy which mostly affects children and young adults. The disease is caused by a mutation in a gene called RPE65 and is passed from generation to generation. Those who have retinal dystrophy suffer from severe vision problems, starting with night blindness and potentially leading to total loss of sight.
According to Spark, Luxturna is not a cure but a way to halt the disease from spreading. The therapy is received via an injection to each eye.
“Gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses,” states Dr. Scott Gottlieb, the FDA’s commissioner.
According to Gottlieb, the agency will roll out guidelines in 2018 to accelerate the approval and evaluation of gene therapy treatments for other diseases.
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