The therapy is the first type approved by the FDA that targets a disease caused by mutations in a specific gene.
The wait is over and Spark Therapeutics announced the price tag for its first-of-its-kind gene therapy for eye disease -- $850,000 for both eyes.
The U.S. Food and Drug Administration approved Luxturna (voretigene neparvovec), a gene therapy for a rare, genetic form of blindness, days before Christmas. The therapy is the first type approved by the FDA that targets a disease caused by mutations in a specific gene. Luxturna is approved for the treatment of pediatric and adult patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. The disease can lead to vision loss and may cause complete blindness in certain patients.
This morning, Pennsylvania-based Spark revealed the wholesale cost of the treatment as well as three new payer programs, including an outcomes-based rebate arrangement, aimed at easing the burden of cost. In its announcement, Spark said it reached an agreement with Harvard Pilgrim and affiliated of Express Scripts to make the gene therapy available under the different pricing programs.
“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,” Jeffrey D. Marrazzo, chief executive officer of Spark Therapeutics, said in a statement. “We believe that access to therapy is a shared responsibility among Spark Therapeutics, payers, health benefit providers, physicians and treatment centers. We have been working with stakeholders across the health care 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.”
The cost for the treatment came in at the low-end of speculation by analysts, who predicted the gene therapy could have a price as much as $1 million, or $500,000 per eye. Still at $850,000, Luxturna is the most expensive therapy in the United States. Biallelic RPE65 mutation-associated retinal dystrophy affects between 1,000 and 2,000 patients in the United States.
Steve Miller, Express Scripts chief medical officer, told CNBC that although expensive Luxturna is responsibly priced by Spark.
“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,” Miller said in his CNBC interview.
For the past several months, Marrazzo said Spark has been working with health insurers to create “innovative pathways for access” to Luxturna that can serve as models for other down-the-road gene therapies. In its announcement this morning, Spark said in addition to the outcomes-based program, the company offered an innovative contracting model that reduces the traditional burdens of “buy and bill” programs that hospitals and other facilities often employ. The company said it has submitted a proposal to CMS under which payments for the gene therapy can be made over time.
“Our work is not done, but we believe that the offerings we are announcing today will help ensure that eligible U.S. patients have the coverage and financial support they need to gain access to both Luxturna and the specialized medical care required to deliver the product at treatment centers,” Marrazzo added.
Luxturna uses a naturally occurring adeno-associated virus (AAV) that has been modified using recombinant DNA techniques to deliver a normal copy of the RPE65 gene directly to retinal cells. The cells then produce the normal protein that converts light to an electrical signal in the retina to restore patient’s vision loss. In clinical trials, patients who received the therapy had significant improvement in light levels in both eyes. Long-term results are not yet available, but so far the effects of the treatment appear to be lasting.
