Luxturna: FDA documents reveal the value of a costly gene therapy. Issue 4 (April 2019)

• Record Type: Journal Article
• Authors: Darrow, Jonathan J.
• Is Part Of: Drug discovery today. Volume 24:Issue 4(2019)
• Page Start: 949
• Page End: 954
• Publication Date: 2019-04
• Subjects: Drugs -- Design -- Periodicals; Drugs -- Research -- Periodicals; 615.1
• DOI: 10.1016/j.drudis.2019.01.019
• Languages: English
• ISSNs: 1359-6446

Abstract

Highlights: Voretigene neparvovec-rzyl has not been shown to cure most patients. Safety concerns of voretigene neparvovec-rzyl are unlike most medicines. Total societal costs of voretigene neparvovec-rzyl exceed the $850, 000 list price. 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$850 000. 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$850 000 figure.