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New gene therapy for inherited blindness could cost patients up to $1m per treatment

A new gene therapy for the treatment of Leber’s congenital amaurosis (LCA) is predicted to be priced at somewhere between $650,000 and $1,000,000 per patient per treatment, sparking considerable debate in the gene therapy market. According to Wall St. analysts, the new gene therapy for LCA may earn up to $200M in revenues in its first three years however, reimbursement decisions by payors in the US, and by national health systems in the EU, will undoubtedly shape the commercial landscape and future market for both the drug itself, and for the wider field of gene therapy.


The LCA treatment, to be marketed as “Luxturna”, was developed by Spark Therapeutics (NASDAQ:ONCE), a gene therapy company based in Philadelphia, USA. In October of this year, the company announced that the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee unanimously recommended (16-0) approval of Luxturna (voretigene neparvovec), as a “potential one-time gene therapy, for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD).” According to Spark, somewhere between one and two thousand patients in the US are understood to suffer from the disorder. Recommendation of Luxturna, previously “SPK-RPE65” was secured on the company’s Phase III clinical trial data, which included data from an intent-to-treat population randomized over a 12-month time period. According to Spark, the clinical trial results showed “a statistically significant and clinically meaningful difference between intervention (n=21) and control participants (n=10) at one year, per the clinical trial’s primary endpoint, mean bilateral multi-luminance mobility testing (MLMT) score change (difference of 1.6; 95% CI, 0.72, 2.41; p=0.001).” The company additionally announced that treated patients had a “marked difference compared to control participants across the first two secondary endpoints: full-field light sensitivity threshold (FST) testing averaged over both eyes (p=0.001) and the mobility test score change for the first injected eye (p=0.001).”


The exact price for Luxturna is not expected to be announced until formal approval by the FDA is secured, potentially scheduled for mid-January of 2018. However, pricing such a therapy for the market is not a trivial task. Justifications can be provided on multiple levels, ranging from the value of funds invested to reach the market, to the value accruing to a patient in the absence of any other treatment. As LCA can strike patients at a very young age, a one time treatment that definitively cures the disease may provide an almost incalculable benefit over several decades of life. However, not all patients respond identically to any given treatment – for non-responders, an upfront payment of $1M would not provide a beneficial investment. There may also be patients that fall between both extremes, experiencing only a partial but incomplete “cure” – should such patients pay the full amount? In the EU, health budgets are often fixed and pharmacoeconomic exercises to allocate scarce resources can at times present a blunt instrument, invariably producing hard cases. The binary alternatives of providing or with-holding treatment in respect of blindness may be extremely challenging to price, and even more challenging to defend. It is to be celebrated that these choices can now be made, given the very long and winding path it has taken to bring such therapies to reality. However, a new set of challenges will now need to be met in order to insure that the benefits of such technologies reach the widest number of patients. Industry has certainly invested significant resources however, in the majority of cases that have created gene therapy opportunities, the technology and the champions that drove the technology forward, arose in and were funded by the public sector. Navigating such pricing territory will be difficult – if such treatments become available to only certain segments of society, the underlying model for public investment in scientific research may ultimately suffer.