The FDA passed along their nod of approval for the sought after gene therapy treatment, Luxturna, reported Engadget. This will be used to help aid those made blind by a rare eye disease. While this is a huge advancement and gene therapy is promising, the price tag is exceptionally high, landing at a whopping $850,000 to treat both eyes. While extreme, many anticipated it would actually be much higher.
The company who developed Luxturna, Spark Therapeutics, is making efforts to make the treatment more accessible, knowing fully well that it’s an extreme cost. One of their plans is to rebate the expenses if the treatment doesn’t significantly give the client visual improvement. Spark also made arrangements for insurers to buy the treatment directly, to avoid any upscaling mark-ups of expenses. They hope it will benefit both the insurers and the treatment centers. Additionally, they are aiming to have some type of payment plan so the treatment can be paid over a longer period of time. Spark is well aware of the situation they are in, and they are making efforts to make this treatment available to the every day patient. Announcements from the company will be made in the upcoming months with more details.
Gene therapy is the hot ticket right now for many rare disease patients. Like other gene therapies, Luxturna replaces the mutated gene, more specifically the retinal dystrophy with a healthy gene. This then allows the eye to properly function and convert light into electrical signals. Right now, there about roughly 1,000-2,000 Americans affected by RPE65-associated Leber Congenital Amaurosis (LCA), which you can learn more about here.
While it’s an amazing procedure and excitement grows to see how many patients it will help, time will tell if it stays around. When treatments for rare-disease are highly priced, it pulls the success rate lower. Recently, in Europe there was an approved gene therapy for rare disease, yet it’s $1 million expense forced it to quickly flop. We can only hope Luxturna can stand it’s ground and that Spark keeps pushing for ways to make it accessible to those who desperately need it.
