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Spark Therapeutics Hopes to Cure a Rare Blindness for $850,000 a Pop

Small biotech firm Spark Therapeutics ONCE recently announced a new gene therapy that is designed to treat a rare, inherited retinal disease that can cause blindness. Approved by the FDA, Luxturna costs $425,000 per eye, or $850,000 for both, though Spark did reveal payment and access programs to help with the astronomical price tag.

The company said that it is exploring payments for Luxturna by installments, as well as tying payments to how well the therapy works. For instance, Spark wants to offer discounts based on whether or not the drug initially works and remains effective; there are roughly 1,000 to 2,000 patients in the U.S. who suffer from this kind of inherited retinal disease caused by a mutant gene.

Spark is also proposing bypassing hospitals and healthcare providers and selling directly to insurance companies and specialty pharmacies. According to Bloomberg, the company is already in talks with Express Scripts ESRX on such a deal.

And, the pharmacy benefits manager looks to be a big proponent of Luxturna as well. Steve Miller, Express’ chief medical officer, said that "Many people were anticipating this [retinal disease treatment] would be more than a million dollars…In the end, this is a revolutionary product, and I think in most plans this will be covered.”

In a phone interview with Bloomberg, Spark CEO Jeff Marrazzo said that “We believe that this price reflects not only the breakthrough, life-altering value of one-time Luxturna, but it will enable us to continue to invest and build on the revolutionary science that supports not only Luxturna but the rest of our pipeline.”

How is Luxturna delivered?

Since Luxturna is a gene therapy, it involves the delivery of a healthy copy of a gene, using an altered virus, in order to make up for one that is deficient and causes a disease, in this case, retinal dystrophy. Luxturna is delivered to patients just once.

In clinical studies, patients’ sight was examined based on their ability to navigate an obstacle course in varying light levels, and those who received the treatment showed huge signs of improvement in doing so at low light after a one-year period, notes CNBC. They also described being able to read again and seeing snowflakes for the first time to a panel of FDA advisers.

Shares of ONCE closed up 4.6% to $55.76 per share.

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