AP
In this Oct. 4, 2017 photo, Dr. Albert Maguire checks the eyes of Misa Kaabali, 8, at the Children's Hospital of Philadelphia. Misa was 4 years old when he received his gene therapy treatment.
A US Food and Drug Administration (FDA) advisory committee just gave a critical recommendation for a cutting-edge gene therapy.
The treatment, called Luxturna, treats Leber congenital amaurosis, a hereditary form of blindness. It's been tested in people with the condition who specifically have a faulty RPE65 gene.
On Thursday, an FDA advisory committee panel voted 16-0 to recommend the drug for approval.
The FDA can take the panel's recommendation into consideration but doesn't necessarily rule the same way. The company is expected to get a decision from the FDA by January.
Here's what you need to know about the drug:
- Leber congenital amaurosis is caused by a gene defect doesn't lead the retina to make a key protein.
- The one-time treatment is injected into the retina of the eye. From there, a virus carrying the corrected gene can get to work replacing the faulty gene and start producing the protein.
- Within a month, the benefits of the treatment start to appear.
- In a phase 3 trial of 20 patients injected with the gene therapy and 10 in the control group, 13 were able to navigate a mobility maze used to gauge vision at the lowest light level a year after treatment. No one in the control group was able to pass through the maze at this light level, leading investigators to conclude that the therapy improved visibility, light sensitivity, and the visual field participants had.
- The treatment, if approved, likely won't be cheap. The first gene therapy the FDA approved costs $475,000, while some gene therapies approved in Europe carry $1 million price tags.