A patient advocacy group criticized the hefty price tag—around $850,000—of a new treatment for blindness.
FDA approved voretigene neparvovec-rzyl (Luxturna, Spark Therapeutics), a one-time gene therapy for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, earlier this month.
Luxturna is the first FDA-approved gene therapy for a genetic disease, the first and only pharmacologic treatment for an inherited retinal disease (IRD) and the first adeno-associated virus (AAV) vector gene therapy approved in the United States, according to Spark Therapeutics.
FDA’s approval of Luxturna “represents a true paradigm shift for physicians caring for patients with hereditary retinal disease caused by biallelic RPE65 mutations, who up until now have had no pharmacologic treatment options,” said Alex V. Levin, MD, MHSc, pediatric ophthalmologist and chief of the Wills Eye Pediatric Ophthalmology and Ocular Genetics Service in Philadelphia, in a statement from Spark Therapeutics.
While Luxturna is a novel drug, its price tag is controversial. “For patients who are underinsured or are insured through government programs such as Medicare and Medicaid, Spark plans to support independent Patient Assistance Programs that may help cover their drug and treatment costs,” Spark said in a statement.
In addition, Spark will offer rebates and installment payments to "help ensure patients' individual financial situation does not hinder access to treatment," Monique da Silva, a spokesperson for Spark, told CNN.
However, “Spark Therapeutics is charging as much for Luxturna as they think they can get away with. Our system cannot handle unjustified prices like this, and the new payment models…are merely a way to disguise a price that is simply too high,” said David Mitchell, president and founder of Patients For Affordable Drugs, in a statement.
"Spark has been very purposeful in its effort to be responsible and responsive in its approach to ensuring this therapy is accessible for patients," says Ellen Drazen, Express Scripts spokesperson. "They approached Express Scripts pre-launch to brainstorm alternative contracting and distribution solutions to balance the needs of patients, payers and providers.
"Our own non-traditional distribution model—offering access through either Accredo or CuraScript—which we have leveraged successfully with other high cost therapies such as Spinraza, reduces the risk providers and hospitals have in keeping expensive inventory in their possession for such a limited patient population, and can help payers manage excessive mark-ups on the product that create substantial additional costs for payers," Drazen says. "As the promise of gene therapy becomes a reality, we believe these non-traditional payment and distribution models will lay a strong foundation from which we can build upon to help put this therapies within reach of the patients who need them."
Luxturna, which will be available in the first quarter of this year, will be administered at selected treatment centers in the US by leading retinal surgeons, who will receive surgical training provided by Spark.