At a time when rare disease drugmakers routinely strike up criticism with their pricing decisions, Spark Therapeutics is surely prepping a pitch for its newly approved Luxturna. On Tuesday, the FDA signed off on the gene therapy, among the world's first, to treat an inherited form of vision loss that can result in blindness.
Luxturna is now Spark's only approved med and works by delivering a gene called RPE65 into a patient's retinal cells, which then produce a protein to restore vision loss. The one-time treatment uses a naturally occurring adeno-associated virus to deliver the gene. According to the FDA, Luxturna is the first U.S. gene therapy that targets a disease caused by specific mutations.
Spark isn't disclosing price until January, but analysts have suggested a potential $1 million price tag for the one-time treatment. Last month, when reporting its third-quarter results, the company said the med offers a "a value in excess" of $1 million, Evercore ISI analyst Steven Breazzano wrote in a note.
"Luxturna is expected to be available for administration in select treatment centers late in the first quarter of 2018," a spokesperson told FiercePharma. According to Breazzano, the company has touted plans to set up three or four "centers of excellence" for the launch, which could grow to 10 over time.
As of its third-quarter report, the company hadn't said how many patients it's identified for potential treatment, according to the analyst.
It remains to be seen how payers will react. In recent years, some superpricey meds have struggled due to lack of demand, payer restrictions or both. UniQure, for instance, had to discontinue its $1 million gene therapy Glybera in Europe, while GlaxoSmithKline recently said it's exploring a sale for its rare disease portfolio. The company's gene therapy Strimvelis has only made its way to a few patients since launch.
With those factors considered, it'll be up to Spark to convince payers and the medical community its med is worth the price. Consensus estimates for sales are $75 million for 2018, Breazzano noted, which implies treatment of 75 patients at $1 million or 50 patients at $1.5 million. Evercore ISI predicted $381 million in sales for the med in 2019.
Analysts with Barclays noted that Spark received a rare pediatric disease priority review voucher with the nod. The vouchers, which drugmakers use to speed up agency reviews of important new meds, have sold for $125 million to $130 million in resale lately.
While the approval marks a first for the FDA, the agency and the medical community already entered a new age for cancer treatment this year when Novartis' Kymriah and Gilead's Yescarta, two CAR-T treatments for rare cancers, won their marketing nods. Kymriah costs $475,000 and Yescarta costs $373,000 before discounts; both are one-time treatments.
In a statement Tuesday, FDA Commissioner Scott Gottlieb said the approval "reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases." Gottlieb added he believes gene therapy "will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses."