A gene therapy for a retinal dystrophy disorder will cost $850,000


The drug is intended for patients with retinal dystrophy due to a mutation of the RPE65 gene. This rare genetic defect causes severe visual impairment beginning in infancy and can eventually lead to blindness. About 6,000 individuals in the world have it. Luxturna is a liquid that is injected directly into the eye with a microscopic needle during a surgical procedure.


The drug will be available in the United States in early spring, with an estimated 1,000 to 2,000 people in the US standing to benefit from this treatment. We have to observe that most patients would need treatment for both eyes, so the total price will be $850,000. The company also stated that its proposal to the Centers for Medicare and Medicaid Services, the government program that covers about 100 million Americans, would allow installment payments for the drug. “A non-traditional payment and distribution model is necessary to ensure needs of all parties, patients, payers and providers , are addressed”, said a spokeswoman for Express Scripts. In the clinical trial demonstrated a gain in functional vision.


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