Successful Eye Treatment Gene Therapy May Get FDA Approval
Next year, there could be an eye treatment employing gene therapy to treat a hereditary eye disorder if its developer gets FDA approval, says Reuters.
Inherited retinal dystrophies (IRDs), an illness that affects the eye, sometimes leading to blindness, was treated successfully by Spark Therapeutics, developer of the gene therapy in Phase 3 clinical trials.
Patients of the improved treatment could move more easily in dim environments, according to HNGN.
Gene therapy means replacing mutated genes with healthy ones, which ought to be a "one-time fix". Yet earlier trials undertaken by other scientists proved that the impact lasted only for a year or several years, according to The New York Times
"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.
Hence, the treatment, named the SPK-RPE65, got closer to becoming the first eye treatment gene therapy that got FDA approval in the U.S. Till now, gene therapy treatment has never won an FDA approval for medical purposes.
"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 explained. His announcement also boosted the company's shares by 50.2 percent on Monday.
By getting approval in 2016, the therapy will cost $100,000 per patient every year. Cowen & Co. analysts pointed out that it might cost $65 per share, while J.P. Morgan analysts judge that there could be "peak global sales at $600 million", according to Reuters.
