Luxturna gene therapy approved for vision loss

Luxturna gene therapy approved for vision loss

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.

A landmark gene therapy from Spark Therapeutics to treat a rare, inherited form of blindness will cost $850,000. The company estimates the condition affects between 1,000 and 2,000 people in the US.

Gene therapy is not alone in commanding staggering sums, particularly when it comes to treatments for rare diseases.

Harvard Pilgrim and Express Scripts, the country's largest pharmacy benefits manager, have also agreed to a new contracting arrangement with Spark that guarantees patient access to Luxturna in exchange for reducing the financial burden and risk for treatment providers and insurers.

Dr Peter Bach, director of a policy centre at Memorial Sloan Kettering Cancer Center in NY quoted regarding the issue, "The company very cleverly convinced everyone that they were going to charge a million dollars, so now they are being credited for being reasonable".

"We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients", said Jeffrey Marrazzo in an interview Wednesday.

But even compared to other one-time gene therapies Luxturna is still an outlier. The manufacturers say the price is a bargain.

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.

The drug is intended for patients with retinal dystrophy due to a mutation of the RPE65 gene.

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That makes the treatment, called Luxturna and made by Spark Therapeutics, the most expensive medicine sold in the USA, ranked by sticker price.

Spark Therapeutics CEO Jeff Marrazzo co-authored a recent blog post in the journal Health Affairs that said "the current payment system - accustomed to ongoing treatment of chronic diseases - creates significant challenges" for gene therapies and other treatments.

Spark said it is also discussing a proposal in which insurers would pay for the drug in installments over several years.

The company said it had agreed bespoke deals with U.S. insurers, which cover the cost of most USA prescriptions, and they will get a refund if the drug doesn't work as expected.

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 United States would cost. And the company is also working on covering patients' out of pocket costs as part of the overall price.

Hardly anyone pays the full price of drugs out of pocket, however.

Steve Miller, chief medical officer at Express Scripts, said: "For one-time therapies like Luxturna, we believe non-traditional payment and distribution models are needed".

"Luxturna offers hope to hundreds of patients", said Patients For Affordable Drugs' Mitchell. "The answer to that is certainly not the $850,000 price tag announced today", he added.

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