The Food and Drug Administration approved the first genetically modified treatment to cure a rare inherited disease.
This therapy is called Luxturna, a modified virus that transmits a health gene into the eyes of people born with retinal dystrophy, a condition that destroys cells in the retina that is needed for healthy eyesight, NPR reports.
In tests conducted, patients who received the gene therapy often produced dramatic results, restoring their vision enough to see things they never could before, like the stars, the moon, fireworks and even their parents’ faces.
The treatment also allowed patients to do many things they had been unable to do, like read, play sports, ride bicycles, or go outside at night alone.
Scott Gottlieb, FDA Commissioner, said,
Today’s approval marks another first in the field of gene therapy. This milestone reinforces the potential of this breakthrough approach in treating a wide range of challenging diseases.
This approval is the latest development in the fast-paced field of gene therapy, which was stuck in disappointments and failures for years.
In August, the FDA greenlit Kymriah, the first gene therapy product able to treat a specific kind of leukemia. A second genetic treatment called Yescarta was approved for a type of lymphoma in October.
Spark Therapeutics, which manufactures Luxturna, has not disclosed how much it will charge for this treatment. There is an expectation that it will cost as much as $1 million for each patient.
Dr. Peter Bach, director of the Center for Health Policy and Outcomes at the Memorial Sloan Kettering Cancer Center, said, “I think the price tag will be enormous — 20 or 30 times the annual wages of the typical American.” He added, “For rare conditions like this, the question we need to ask ourselves is exactly how much wealth should be transferred from society to the investors in these companies.”
The FDA approved the treatment after unanimous recommendations for it by an advisory committee convened in October.