Update: Gene Therapy for Eye Disease Becomes Most Expensive Drug Priced at $850,000

Spark Therapeutics’ Luxturna, a pioneering gene therapy that reverses vision loss in patients with an inherited form of blindness, was approved by the FDA just before the regulator broke for the holidays. Now, the Philadelphia-based company has announced the list price of the gene therapy at $425,000 per eye, or $850,000 for the pair, making it the most expensive drug in the US.

But according to Forbes, analysts from investment bank Jeffries originally predicted the cost of the gene therapy would be set around $1 million due to the curative nature of the treatment and the fact that no other therapies exist for retinal dystrophy. It’s also worth remembering that the bulk of the $850,000 price tag will likely be absorbed by insurance companies capable of negotiating deep discounts on drug prices.

At the same time, Spark has announced new initiatives to help eligible patients gain access to Luxturna, and help offset the cost for payers. The company has established an outcomes-based rebate agreement with the not-for-profit health services company, Harvard Pilgrim, whereby the insurer would receive rebates if and when patients fail to see short-term efficacy (30-90 days) and longer-term durability (30 months) of the gene therapy.

“To help ensure eligible patients have access to Luxturna, we are striving to bring the same level of innovation applied in development to the delivery of, and access to, this product,” said Jeffrey D. Marrazzo, chief executive officer of Spark Therapeutics. “We have been working with stakeholders across the healthcare sector to help ensure that appropriate patients have access to a product that challenges all of the current conventions of how patients are treated, how products are delivered and how payments are handled.”

In addition to the outcomes-based rebate arrangement – which Spark hopes to offer to other select payors – a contracting model has been established which would allow payers to buy Luxturna directly without going through a hospital or other healthcare center. This deal has already been made with Express Scripts, and should help payers manage treatment costs while removing the burden on hospitals of purchasing and storing a high-priced drug.

High prices are common among rare disease drugs, particularly those capable of having a curative effect. What’s more, Luxturna is designed to be a one-time treatment which requires that Spark recoup development costs through individual, one-time payments.

As many as 2,000 patients in the US will be eligible for treatment with Luxturna, however they will be required to have a confirmed biallelic RPE65 mutation before being considered for treatment with the gene therapy. Spark and patient advocacy groups are encouraging patients with retinal dystrophy to get their genetic diagnosis to confirm their eligibility.