Spark Therapeutics' Luxturna, the first gene therapy to gain FDA approval for the treatment of a rare, inherited retinal disease that can lead to blindness, will cost patients $850,000 for a one-time treatment, or $425,000 per eye, CNBC reports.
The drug was approved for use in the U.S. in December. The Philadelphia-based biotechnology company would deliver the treatment once, fueling the price-controversy surrounding the costly arena of gene therapy.
Gene therapy uses a modified virus to deliver a healthy copy of a gene to replace a deficient one — specifically, the gene that causes a disease. Luxturna injects copies of a normal version of the gene RPE65 into a patient's eye. That gene is responsible for producing a protein integral to maintaining light receptors. Patients who have the rare genetic disease have a mutated version of RPE65, which slowly causes vision loss and in most cases, almost all patients end up completely blind.
Wall Street predicted Luxturna's price tag would ring in around $1 million, so Spark's ultimate decision falls short of that mark.
"It's wildly expensive but, to be very frank, I think they've priced it what I'll call responsibly," said Steve Miller, MD, CMO of Express Scripts, which is partnering with Spark to distribute and negotiate reimbursement for Luxturna. "The product is just phenomenally innovative, and we've been talking about gene therapy for over 20 years. We're now at the threshold of having gene therapy reaching patients."
The company also laid out several potential payment models that aim to increase patients' access to the drug and reduce risk for insurers and hospitals, according to CNBC.