A first-of-its-kind genetic treatment for blindness will cost $850,000, less than the $1 million price tag that had been expected, but still among the most expensive medicines in the world.
Spark Therapeutics said Wednesday it decided on the lower price for Luxturna after hearing concerns from health insurers about their ability to cover the injectable treatment.
Consternation over skyrocketing drug prices, especially in the U.S., has led to intense scrutiny from patients, Congress, insurers and hospitals.
“We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients,” said CEO Jeffrey Marrazzo.
Luxturna is still significantly more expensive than nearly every other medicine on the global market, including two other gene therapies approved earlier last year in the U.S. Approved last month, Luxturna is the nation’s first gene therapy for an inherited disease. It can improve the vision of those with a rare form of blindness that is estimated to affect just a few thousand people in the U.S.
Luxturna is an injection – one for each eye – that replaces a defective gene in the retina, tissue at the back of the eye that converts light into electric signals that produce vision. The therapy will cost $425,000 per injection.
The treatment is part of an emerging field of medicine that could produce dozens of new gene-targeting medications in the next few years.
These therapies are generally intended to be taken once, a fact that drug developers argue sets them apart from traditional drugs taken for months or years.
But even compared to other one-time gene therapies, Luxturna is still an outlier. Two customized gene therapies for blood cancer approved last year are priced at $373,000 and $475,000, respectively.
The estimate by the non-profit Institute for Clinical and Economic Review assumes the drug would maintain patients’ vision for 10 years. However, Spark expects the drug’s effect to be long-lasting, if not lifelong, though it has only tracked patients for about four years.
At least one gene therapy sold overseas has already crossed the $1 million price threshold, a treatment for a rare protein disorder launched in Europe. Manufacturer uniQure stopped selling the therapy last year after seeing a lack of demand. It was never approved in the U.S.
Like most prescription medicines in the U.S., most of the immediate costs of Luxturna will be borne by insurers – not patients – including private plans and government programs.