On Tuesday, a U.S. Food and Drug Administration Review Committee published a document that may just mean a big moment in medicine is just around the corner. The committee found that an experimental gene therapy for a rare inherited form of blindness would be able to improve vision. At least in the short term. If approved, the technique would be the first gene therapy for an inherited disease approved in the United States.

The drug is being reviewed two days before a meeting of outside experts who are going to recommend to the FDA whether or not the drug should be approved. The reviewers note that the drug meets the criteria for approval, so it’s hoped that it will go through. But there is no long-term data to determine what this drug might mean in the future.

A study was conducted that involved 21 patients with the inherited blindness condition known as Leber congenital aumaurosis. The condition is triggered by a gene mutation. Of the 21 patients, 11 experienced significant vision improvement. 93% of the participants experienced at least some improvement. But overall, this isn’t a sure-fire treatment. The drug works in a really interesting way. It delivers a correct copy of the mutated gene to the retinal cells. Which then restores a person’s ability to produce the deficient enzyme that causes the condition. This is interesting because it makes you wonder for how long the correct gene will be delivered.


This is unknown because there is no long-term data. A panel will review and consider the possibility that patients may need multiple treatments over time. This is controversial as this treatment was originally billed as a “one-time” treatment. But that doesn’t mean it doesn’t work. Or at least help some patients. How much it helps, and to what extent is yet to be determined. This is definitely something that the FDA is going to want more information on.

If approved, the therapy itself could help between 1,000 and 2,000 people in the United States with this inherited retinal disease. But will that be enough? I don’t mean to make it sound like 2,000 people isn’t enough to warrant the treatment from being available. But it does make you wonder if it will go through. The number of people it is going to help shouldn’t impact the FDA’s decision, but it’s effectiveness will. Furthermore, is a study of 21 people enough to determine what impacts this drug will have on patients and the medical world?

I do hope that this is a step in the direction of finding more solutions for these kinds of issues. And, I certainly hope that it is effective. I think that the numbers are low, so I wonder if the FDA will think about this as well. Another thing that is kind of concerning is how much it would cost. While no numbers are known at this point, other gene therapies are likely to cost hundreds of thousands of dollars. I wonder if people are even going to be able to afford the therapy that could ultimately bring their vision back?


By Staff Writer

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