FDA advisers on Thursday, Oct. 12, will consider whether to recommend the approval of said therapy, which has improved the vision of some people with hereditary blindness.
The therapy is called Luxturna, made by Philadelphia-based Spark Therapeutics. It does not provide patients with 20/20 vision nor does it work on everybody, but a study has found that Luxturna improved the vision of nearly all who participated in the trials, and "seemed safe," as Associated Press reports via ABC News.
Many of the participants in these trials are now no longer legally blind, and some have since gained independence.
Luxturna Gene Therapy
Luxturna would be the first gene therapy to become available in the United States for an inherited disease, and the first one where a corrective gene is directly given to the patient. The therapy has been tested on patients with Leber congenital amaurosis, which makes them unable to produce a protein needed in the retina because of an impaired RPE65 gene. They're still capable of seeing blurred shapes and lights — initially — until they lose their vision altogether.
Parents who carry the gene might not even know that they have it, and it can remain undetected for generations — that is, until a kid is born blind, caused by two copies of the flawed gene. It's an unfortunate scenario, but one Luxturna might be able to fix.
"It's exciting," said Dr. Paul Yang, an eye specialist at the Oregon Health & Science University who is testing gene therapies for other companies. In some cases, gene therapy can be a cure, although it remains uncertain just how long the benefits of the treatment last. It is, however, much, much better than nothing at all.
"There's nothing else for these kids," said Yang.
FDA Gene Therapy Approval Deadline
The FDA must decide before Jan. 18, 2018 whether to approve Luxturna, but there's a problem: the therapy is a highly expensive procedure. One therapy sold in Europe cost $1 million and was performed on only one or two patients.
For its part, Spark Therapeutics says it has talked with insurers, claiming that "there is a clear path for it to be reimbursed one time per eye."
Not all forms of blindness are caused by the aforementioned flawed gene, however, as there are over 260 genes that could result in retinal disorders. This means that while Luxturna helps to improve vision, it's only applicable for people with the flawed RPE65 gene instead of a more specific disease.