- Spark Therapeutics has secured marketing authorization from the European Commission for its gene therapy Luxturna, OK'd for a rare inherited type of blindness. The thumbs up makes Luxturna the first gene therapy approved for a genetic disease in both the U.S. and Europe.
- Luxturna works by replacing a dysfunctional gene in patients with RPE65-mediated retinal dystrophy. Approval hinged on clinical trials which showed treated patients had significant improvements in functional sight after receiving the therapy. The EMA's Committee for Medicinal Products for Human Use (CHMP) recommended Luxturna's approval in September.
- Reimbursement is going to be a major challenge for one-time therapies like Spark's. The biotech set a list price of $425,000 per Luxturna injection in the U.S. Novartis, which licensed rights to the therapy in Europe, hasn't disclosed an EU price, but negotiations with health technology assessment bodies like the U.K.'s National Institute for Health and Care Excellence will likely be a pinch point.
Novartis picked up ex-U.S. rights to Luxturna (voretigene neparvovec-rzyl) in a January deal that paid Spark $105 million upfront and lined up another $65 million in potential milestone payments. The deal allows the smaller biotech to focus resources on the U.S., where it snagged approval last December and has already launched Luxturna. Spark will manufacture and supply the therapy to Novartis.
Despite the burden of the inherited retinal disease on patients and affected families, securing reimbursement could still be a point of contention.
The Institute for Clinical and Economic Review, an organization well known for objecting to high therapeutic product costs, determined the $850,000 list price per patient is well above cost-effectiveness thresholds. While Spark has inked agreements to get Luxturna covered in the U.S., it's too early yet to say what national health authorities across Europe will demand in the way of discounts.
That challenge will likely persist too, as stakeholders work out the logistics of pricing a potential cure.
Novartis is already aware the process will take some time. "Decisions from national reimbursement bodies on Luxturna for patients with vision loss due to a genetic mutation in both copies of the RPE65 gene are expected in 2019 and 2020," the pharma wrote in a Nov. 23 statement.
IRDs are caused by mutations in any of over 220 different genes. People with IRD caused by mutations in the RPE65 gene often develop night blindness and involuntary back-and-forth eye movements. Almost all will eventually become blind.