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Luxturna Gene Therapy for Blindness Priced at $850K

12 January 2018

It's the first true gene therapy approved in the USA for an inherited genetic disease - vision loss caused by mutations in a gene called RPE65.

Some critics say it is too high, but others have praised the gene therapy pioneers for showing "leadership" with a performance-related reimbursement scheme.

Some patient groups are already pushing back. Food and Drug Administration in December for Luxturna.

Speculation about how Spark would price the one-time therapy has been ongoing for several years now.

Still, the price tag is higher than any other such treatments on the market.

Today, finally, is the beginning of another important chapter in the gene therapy story.

Luxturna could potentially become available free on the NHS in the United Kingdom after being submitted for approval by the European Medicines Agency (EMA).

"Spark CEO Jeffrey Marrazzo's comments, implying that $850,000 represents something other than greed, are absurd and obscene", Maybarduk said.

The biotech said it has reached an agreement in principle with New England-based insurer Harvard Pilgrim that would base pricing on outcomes. Such arrangements could include Spark paying rebates if patient outcomes fail to meet specified thresholds for efficacy in both the short term (30-90 days) and long term (30 months). And the company is also working on covering patients' out of pocket costs as part of the overall price.

Traditional models usually require the hospital or institution administering a specialty medication to purchase it upfront and then charge the payer.

Kymriah is a leukemia treatment made by pharmaceutical giant Novartis. Current government price reporting requirements do not allow for payment models of this type, but Spark is hoping to change the paradigm. In this gene therapy named Luxturna, there is a 45-minute operation gets performed by the experts.

Luxturna's price is less than what many analysts and outside investors expected, and commercially insured patients likely won't have to pay for the treatment. "We are encouraged by CMS' willingness to engage with us in exploring a new model".

Express Scripts is partnering with Spark for distribution of Luxturna, Terrell reports.

This restores the patient's ability to make the missing enzyme - dramatically improving their eyesight and restoring a large degree of independence, although not offering 20/20 vision.

After the FDA approval, speculation turned to its price. Gene therapies can be one-off doses that in effect, cure a specific disease. Plus, it is a one-time treatment.

These new prospective models are situated inside a greater debate that is now occurring as more of these breakthrough gene therapies become real.

In Europe, where the first two gene therapies have been rolled out to a mere handful of patients in single payer systems, the failure to gain acceptance has been a virtual death sentence for manufacturers.

"Our work is not done, but we believe that the offerings we are announcing today will help ensure that eligible USA patients have the coverage and financial support they need to gain access to both Luxturna and the specialized medical care required to deliver the product at treatment centers".

Luxturna Gene Therapy for Blindness Priced at $850K