FDA to approve gene therapy for rare form of blindness

gene therapy

The FDA panel said Tuesday in a review posted on its website. It is considering approval but the long-term benefits of the treatment remain unclear. A meeting with advisers who  recommend whether the treatment should be approved is set for Thursday.

Spark Therapeutics the drug developer will face questioning Thursday by U.S. Food and Drug administration medical experts on the company’s pioneering gene therapy that restores vision in patients with a rare hereditary blindness.

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Leber congenital amaurosis retinal disease

The treatment tested 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,

If the FDA gives the green light, around Jan. 12. 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.

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.

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.

Moreover, the agency must announce its final decision on Jan. 18. Scientifically named voretigene neparvovec, Luxturna might cost thousands of dollars. Companies usually announce costs only after their treatments are 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

The FDA may approve the treatment in January, it becomes the first gene therapy in the United States for an inherited disease.

Spark’s treatment, voretigene neparvovec, has received “priority review” by the FDA for 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.

A natural protein that protects the eye from glaucoma

Spark’s treatment is onetime because of concerns that repeat injections could lead to serious immune reactions. “We generally expect a warning that repeat administrations into the same eye should not be done,” Nadeau wrote. 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.