Gene therapy to treat a hereditary disease that causes blindness has received an eye-popping price tag of $850,000.
The drug, named Luxturna, was recently approved by the United States' Food and Drug Administration. It currently has a prohibitive cost, but the medicine may be the first step in the rise of gene therapy as a cure to various diseases and conditions.
What Does Luxturna Do?
Luxturna, as approved by the FDA, specifically targets a retinal dystrophy that is named biallelic RPE65. The disease, which affects up to 2,000 people in the United States, is caused by a genetic mutation that gradually disrupts the cells in the back of the eye forming the retina. This results in patients slowly losing their vision, leading to blindness for which there is no cure.
Luxturna is the first true gene therapy approved by the FDA for an inherited genetic disease. The treatment injects a modified cold virus straight into the patient's eye to deliver corrected genes directly to the retina.
A single treatment of Luxturna almost immediately reversed vision loss in most of the 21 volunteers who have received the drug.
Is Luxturna Too Expensive?
At $850,000 for a treatment of Luxturna, the medicine appears to be grossly expensive. However, in a press release, Spark Therapeutics, the company behind the gene therapy, noted that this is a one-time treatment. In addition, it is working with stakeholders in the healthcare industry, including health insurers, for installment options and rebates.
However, even though the cost is lower than the $1 million price tag that analysts were expecting, there remain concerns that the drug is overpriced.
"The new payment models announced today are merely a way to disguise a price that is simply too high," said Patients For Affordable Drugs founder and president David Mitchell. "Spark Therapeutics is charging as much for Luxturna as they think they can get away with."
Gene Therapy: The Future Of Medicine?
As questions swirl around the cost and effectiveness of gene therapy, it could not be doubted that Luxturna is a breakthrough in the development of this kind of medicine.
Gene therapy, instead of addressing the symptoms of inherited diseases, looks to correct defective genes that form their foundation. In addition to inherent blindness, gene therapy is being developed to cure hereditary deafness, leukemia, sickle cell anemia, and many others diseases.
Luxturna is not quite there yet, as clinical data shows that it does not fully restore vision and it only works for up to four years. However, medical researchers will likely continue working to improve the effectiveness, and hopefully also to bring down the price, of gene therapy.