It's a gradual and cruel fate: an inherited form of vision loss that typically ends in blindness and has had no cure. But for those with mutations in a gene known as RPE65 comes a balm. The FDA in December approved Luxturna, a new gene therapy that can treat the progressive blindness, and on Wednesday, its maker revealed the staggering cost: $425,000 per eye. Pricing it was entering a bit of a brave new world: NBC News reports Luxturna is the first "true gene therapy" that's gotten the green light here for an inherited disease, and unlike other pharmaceutical companies' workhorses—drugs that patients take for weeks, months, or even a lifetime—Luxturna is expensive to create and delivered just once. And as the Washington Post explains, Luxturna isn't necessarily a cure for the up to 2,000 Americans who suffer from the disease.
In trials, it has "worked" for patients for as long as four years (FierceBiotech explains patients are born with poor vision, and their vision doesn't become "normal" with Luxturna but does functionally improve), though there are indications its effects could extend beyond that mark. The Guardian reports that maker Spark Therapeutics had previously tossed out the figure $1 million when discussing the total treatment cost, saying that was a fair amount when you consider the cost of blindness. The Post says it went with the lower figure in part because insurers indicated $500,000 per eye would shrink the pool of those who qualified, and the company is discussing rebate and payment plan options. But one critic tells NBC he wants Spark to release its R&D costs so "analysts, payers, and the public have a basis to assess Spark's decision."