An eye treatment that employs gene therapy in treating a hereditary eye disorder could be available on the market as soon as its developer gets FDA approval next year, Reuters reports.
Spark Therapeutics, developer of the gene therapy, announced that phase 3 clinical trials showed it was successful in treating inherited retinal dystrophies (IRDs), an eye disease that causes blindness. People who received the treatment reportedly had improved vision and were able to move more easily in dim environments.
"We saw substantial restoration of vision in patients who were progressing toward complete blindness," Albert M. Maguire, lead researcher in the study and a professor of ophthalmology at the University of Pennsylvania, said in a press release.
The announcement brought the treatment, called SPK-RPE65, closer to becoming the first eye treatment gene therapy to receive FDA approval in the U.S.
"This is a watershed moment in the long-time pursuit of innovative gene therapy solutions for a range of blinding retinal degenerative diseases," Gordon Gund, chairman of the Foundation Fighting Blindness, said in the press release.
Spark's announcement also made the company's shares soar by 50.2 percent on Monday.
If the treatment gets approval in 2016, it is expected to cost $100,000 per patient annually. Cowen & Co. analysts said it could cost $65 per share, while J.P. Morgan analysts estimate peak global sales at $600 million, according to Reuters.
So far, no gene therapy treatment has ever won an FDA approval for medical purposes. Gene therapy involves replacing mutated genes - those that are responsible for the disease - with healthy genes. In theory, it should be a one-time fix for the disease, but previous similar trials conducted by other researchers showed the effect lasted only for a year or several years, The New York Times reports.
