First Gene Therapy For Blindness Will Cost $850,000 U.S. Dollars

Luxturna, the first gene therapy treatment approved in the United States for a rare, inherited form of blindness, will have a wholesale acquisition cost of $850,000 U.S. dollars per patient, or $425,000 per eye, announced its maker, Spark Therapeutics Inc., on Wednesday, January 3.

“The price reflects the stakeholder considerations we have learned about these past months, including access concerns of patients, families, health care providers and treatment centers,” explains Monique da Silva, a spokesperson for Spark Therapeutics.

While the price ended up lower than the one-million-dollar estimate by U.S. analysts, it’s still among the most expensive treatments in the world.

Luxturna was approved last month by the U.S. Food and Drug Administration (FDA) to treat children and adult patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss and may cause complete blindness in certain patients.

Unlike traditional drugs, which tend to be taken for months or years at a time, gene therapies are intended to be one-off treatments that tackle a disease at its source, repairing faulty DNA so the body can fix itself.

Spark’s Luxturna works by delivering a normal functioning copy of the missing RPE65 gene directly to retinal cells, leading these cells to produce the normal protein that converts light to an electrical signal in the retina to restore the patient’s vision. In clinical trials, the medicine restored eyesight in some patients with severe visual impairment.

Although Luxturna is intended for a relatively rare type of inherited blindness, several companies are developing similar therapies for more common illnesses. Spark targets hemophilia, while others are focused on other blood disorders.

The advent of single-time gene therapies — and of new cancer cell therapies from Novartis and Gilead that are also administered once — has prompted a debate over how much drugmakers should charge for scientific breakthroughs and whether society can afford them.

Prior to this approval, the FDA had greenlit two other single-time gene therapies that re-engineer people’s own immune cells to treat blood cancers.

Both came with hefty price tags: Gilead’s Yescarta, a drug that treats certain types of large B-cell lymphoma, has a price tag of $373,000 dollars;  Novartis’ Kymriah, a leukemia therapy, is similarly expensive at $475,000 dollars.

Spark also announced what it described as “first of their kind” programmes designed to help employers, the government, and patients manage the cost of Luxturna.

The group said it would share the risk of the treatment failing by paying some health insurers a rebate linked to whether the treatment worked in the first 30 to 90 days, and also to whether it was still effective after 30 months.

The drugmaker has reached an agreement in principle to offer the outcomes-based rebate arrangement with Harvard Pilgrim, a Boston-based, non-profit healthcare group which sells health insurance plans in New England, and is also in active discussions with other commercial insurers regarding this offering.


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