Cost of treating rare genetic blindness: $425000 per eye

Spark Therapeutics has set a wholesale acquisition cost of $425,000 per eye for its gene therapy Luxturna, a one-time treatment for a genetic condition that causes blindness.

These therapies are generally meant to be taken once, a fact which drug developers argue sets them apart from traditional drugs taken for months or years.

Spark Therapeutics has divided opinion with its $850,000 price for its Luxturna eye gene therapy, which will make it the most expensive drug in the US. This particular genetic blindness only affects a few thousand people in the U.S., making the market for it rather niche.

Luxterna delivers a normal, non-mutated, copy of the RPE65 gene directly to the cells of the retina. This results in patients slowly losing their vision, leading to blindness for which there is no cure.

A single treatment of Luxturna nearly immediately reversed vision loss in most of the 21 volunteers who have received the drug.

The inherited disorder it treats often leads to blindness, reports CNN's Susan Scutti. For instance, cholesterol-lowering drugs must be taken daily for life and they are taken by millions of people.

The mutation affects both eyes, usually at the same pace, so most patients would need treatment for both eyes, a Spark Therapeutics spokeswoman said.

How does Luxturna's price compare to some of pharma's other superpricey drugs?

The advent of single-time gene therapies - and of new cancer cell therapies from Novartis and Gilead that are also administered once - has prompted a debate over how much drugmakers should charge for scientific breakthroughs and whether society can afford them.

Luxturna, as approved by the FDA, specifically targets a retinal dystrophy that is named biallelic RPE65. That rebate program was made with nonprofit health plan Harvard Pilgrim.

Most prescription drugs in the United States are paid for by insurers, including private plans and government programs like Medicare and Medicaid, which provide health coverage to the poor and elderly.

Consternation over skyrocketing drug prices, especially in the U.S., has led to intense scrutiny from patients, Congress, insurers and hospitals. Steve Miller, Express' chief medical officer, said that "Many people were anticipating this [retinal disease treatment] would be more than a million dollars..."

"The product is just phenomenally innovative, and we've been talking about gene therapy for over 20 years; we're now at the threshold of having gene therapy reaching patients".

Spark will also offer patient assistance programs with the aim that all eligible patients have access to Luxturna. "We've got to figure this out, because - let's be frank - there are going to be more of these drugs coming to the marketplace for even bigger populations". Under the agreement, payers would expedite benefits processing and cap patient out-of-pocket costs at in-network limits.

Some patient groups are already pushing back.

To further allay concerns over the cost of the drug, Philadelphia-based Spark will use unconventional pricing models and schemes with insurers.

"Luxturna offers hope to hundreds of patients. But ... what is a fair price that will maximize affordability and accessibility and provide a reasonable return for the drug?" "The answer to that is certainly not the $850,000 price tag announced today", he added.

"Our health care system is cracking under the strain".