Luxturna: FDA documents reveal the value of a costly gene therapy

Drug Discov Today. 2019 Apr;24(4):949-954. doi: 10.1016/j.drudis.2019.01.019. Epub 2019 Jan 31.

Abstract

In 2017, the US Food and Drug Administration (FDA) approved voretigene neparvovec-rzyl (Luxturna), a gene therapy used to treat a rare form of inherited blindness. Widely described by the media as a curative treatment that 'restores vision', it was priced at US$850000. Although voretigene neparvovec-rzyl represents a substantial therapeutic advance, most reports have failed to adequately describe study outcomes as documented by FDA reviewers. These documents reveal that the drug is not expected to restore normal vision, that only about half of treated patients met the FDA's threshold for minimally meaningful improvement, that improvements might not persist long-term, that the most common measure of visual function was rejected as a primary endpoint after yielding mixed results, and that two patients experienced permanent vision loss. Over US$100 million of additional publicly-funded costs are not evident from the US$850000 figure.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Costs and Cost Analysis
  • Genetic Therapy / economics*
  • Humans
  • Retinal Dystrophies / economics*
  • Retinal Dystrophies / therapy
  • United States
  • United States Food and Drug Administration
  • Vision Disorders / economics*
  • Vision Disorders / therapy
  • cis-trans-Isomerases / deficiency
  • cis-trans-Isomerases / genetics

Substances

  • retinoid isomerohydrolase
  • cis-trans-Isomerases