Price tag on gene therapy for rare form of blindness: $850K

WASHINGTON — A first-of-its kind genetic treatment for blindness will cost $850,000 per patient, making it one of the most expensive medicines in the world and raising questions about the affordability of a coming wave of similar gene-targeting therapies.

The injectable treatment from Spark Therapeutics can improve the eyesight of patients with a rare genetic mutation that affects just a few thousand people in the U.S. Previously there has been no treatment for the condition, which eventually causes complete blindness by adulthood.

Pricing questions have swirled around the treatment due to a number of unusual factors — it is intended to be a one-time treatment, it treats a very small number of patients and represents a medical breakthrough.

Previously, Spark suggested its therapy, Luxturna, could be worth more than $1 million. But the company said Wednesday it decided on the lower price after hearing concerns from health insurers about the affordability of the treatment.

Consternation over skyrocketing drug prices, especially in the U.S., has led to intense scrutiny from patients, politicians, 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, in an interview with The Associated Press.

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.

Pharmaceutical industry critics said the slightly lower cost is a distraction from the ongoing problem of unsustainable drug prices.

“The company very cleverly convinced everyone that they were going to charge a million dollars, so now they are being credited for being reasonable,” said Dr. Peter Bach, director of a policy center at Memorial Sloan Kettering Cancer Center in New York.

Approved last month, Luxturna, is the nation’s first gene therapy for an inherited disease. It requires a 45-minute operation in which a tiny needle delivers a replacement gene to 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 genetargeting medications in the next few years.

Like Luxturna, these therapies are generally intended to be taken once, a fact which drug developers argue sets them apart from traditional drugs taken for months or years. Even compared to other onetime 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.