There’s a new candidate for the list of the world’s most expensive drugs. It’s a form of gene therapy called Luxturna, and it was approved in the U.S. by the FDA last month.

This week the company ended speculation by revealing the price. The one-time injection will cost $425,000 per eye — a total of $850,000 US for the full treatment.

The good news is that the long-promised gene therapy treatments are finally becoming available. The bad news is that they cost more than most families can hope to afford.

“We’ve had these promises for a very long time that we’d have these gene therapies,” said Marc-André Gagnon, a pharmaceutical policy researcher at Carleton University. He told CBC News that what happens with the price of Luxturna will set a precedent for future gene therapies.

“If we set the price at such a high level you open the floodgates and things will get worse from there.”

Luxturna uses a virus to insert a replacement gene into the cells of people born with specific mutations that lead to blindness. The treatment is targeted at fewer than 2,000 people in the U.S. and about 200 in Canada.

These “ultra-rare” conditions begin in infancy and lead to progressive blindness in early adulthood. Luxturna is not considered a cure, and its long-term efficacy has not been established. But in small trials it was shown to improve light sensitivity and vision in some patients.

The treatment is not yet approved in Canada. Spark Therapeutics did not respond to our request for information about whether it will apply for Canadian approval, nor did the company respond to our question about why the drug costs so much.

In the U.S., the company has announced programs to help patients get access to the drug, including instalment plans to spread payments over several years, and some form of rebate for some patients if the treatment is ineffective.

“Spark Therapeutics is charging as much for Luxturna as they think they can get away with. Our system cannot handle unjustified prices like this,” David Mitchell, founder of the U.S.-based Patients for Affordable Drugs, said in a statement.

The first gene therapy to come on the market was Glybera, a one-time injection that treated an ultra-rare blood disorder. It was approved in the EU in 2012 and it was priced at €1 million ($1.5 million).

But the therapy was reportedly sold only once. Last year the company, uniQure, withdrew the drug from the marketplace. Second Opinionreported the Glybera story last April.
Original article at: http://www.cbc.ca/news/health/second-opinion-january-6-2018