The FDA panel approved new gene therapy Luxturna. The treatment for Leber congenital amaurosis (LCA) an inherited retinal disease caused by defects in a gene called RPE65. Parents can unknowingly carry the flawed gene for generations.
Patients with a rare inherited form of blindness could be able to regain their sight if the Food and Drug Administration approves an experimental gene therapy in January.
Spark Therapeutics Inc. is the company which has been testing Luxturna. Potentially the first gene therapy for an inherited disease approved in the United States.
Leber congenital amaurosis retinal disease
Patients born with this abnormality are unable to produce a protein that fuels the retina so it can convert light into signals to the brain. Sometimes they can only see blurry shapes and bright light until they completely lose all sight.
The most common adverse reactions from treatment with Luxturna included eye redness (conjunctival hyperemia), cataract, increased intraocular pressure and retinal tear.
Luxturna also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
Treatment with Luxturna must be done separately in each eye on separate days, with at least six days between surgical procedures. It is administered via subretinal injection by a surgeon experienced in performing intraocular surgery. Patients should be treated with a short course of oral prednisone to limit the potential immune reaction to Luxturna.
Spark’s treatment commercialized under the brand name Luxturna. Spark chief executive officer Jeffrey D. Marrazzo has said the company would not discuss pricing before the FDA approves the product.
Scientifically named voretigene neparvovec, Luxturna might cost thousands of dollars. Companies usually announce costs only after treatments approved. But one gene therapy sold in Europe costs $1 million. Luxturna the second gene therapy sold in the country since the FDA approved in August a treatment that engineer’s patient’s blood cells in a laboratory. Jeff Marrazzo, Spark’s CEO, said he has talked with insurers and they might reimburse the treatment one time per eye.
First gene therapy in United States
Spark’s treatment, voretigene neparvovec vision loss due to RPE65 mutation-associated retinal dystrophy, which affects about 3,500 people in the United States and Europe, and has no approved treatments.
Clinical trials showed the treatment improved vision in patients for up to three years. With observation ongoing. With a safety profile consistent with this type of injection procedure the briefing document said patients followed for 15 years.
The advisory committee will also discuss the appropriate ages to give the therapy to patients. Data showed less benefit in older patients with more advanced disease. FDA advisers may recommend Luxturna for patients, ages 4 to 44, with earlier stages of the disease.
Spark’s treatment is onetime because of concerns that repeat injections could lead to serious immune reactions. Long-term data on eight patients from a Phase 1 trial showed the treatment’s effect continued “to be durable for at least four years after administration,” he said.