Finance

Spark Therapeutics offers medicine for treatment of rare inherited form of blindness

Spark Therapeutics offers medicine for treatment of rare inherited form of blindness

Spark's Luxturna was approved by the U.S. Food and Drug Administration in December for patients who have a genetic mutation in the RPE65 gene, which causes degenerative retinal disease.

Years of debate, speculation and analysis have boiled down to this: Spark $ONCE Therapeutics has set an $850,000 wholesale acquisition cost for the US's first gene therapy - $425,000 per eye damaged by an RPE65 gene mutation.

Anticipating criticism, Spark promised to reimburse patients if the treatment proves not to be effective, and said it expects USA health authorities to authorize payment in stages.

The US Foundation Fighting Blindness estimates there are around 18 therapies in development for other inherited forms of blindness.

Gene therapy can be especially risky and expensive to develop, but the company got tax breaks, Public Citizen noted.

In clinical trials, injections of Luxturna restored eyesight to people with severe visual impairment due to retinal dystrophy. However, "there are very few such patients, and the price of every treatment matters a great deal".

"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.

Doctor charged with having wife killed to protect drug ring
James Kauffman , for the fatal shooting of her mom, who was found dead on May 10, 2012, in her home on the Jersey Shore. But Tyner also said at the time that the raid involved the ongoing investigation into Kauffman's murder.

"If you look at the history of biotech, the first approved products don't necessarily define what the market is", he says. Many investors expected Spark to charge $1 million or more for Luxturna, so the actual price will be considered a bargain by some. Their out-of-pocket cost is based on their individual healthcare insurance plans and duration of treatment. The product, approved in December by the Food and Drug Administration, is the first US -approved gene therapy for an inherited disease.

With two cancer therapies approved previous year, and now a third therapy for vision, 2017 has paved the way for many more that will most certainly be coming down the pike in 2018. That idea would apply to government programs like Medicare and Medicaid, which provide health coverage to the poor and elderly.

"Spark Therapeutics is charging as much for Luxturna as they think they can get away with", said David Mitchell, a patient with incurable blood cancer and the president of Patients For Affordable Drugs. While it's less than the $1 million price tag that analysts had expected, it's still among the most expensive genetic therapies in the world. The Swiss drug maker has pledged to reimburse insurers and patients if the $475,000 drug doesn't work.

Marrazzo's not saying how much he's offering in rebates, but when I asked him why not a full refund for patients who don't respond adequately, he said that's not possible.

From the time that Jeff Marrazzo first started at the helm of upstart Spark Therapeutics about 5 years ago, he's been thinking about what the first gene therapy in the USA would cost.

After the FDA approval, speculation turned to its price. The pharmaceutical company said that is now negotiating with other insurers for similar payment schemes linked to treatment outcomes. "Our system can not handle unjustified prices like this, and the new payment models announced today are merely a way to disguise a price that is simply too high".


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