Luxturna: FDA documents reveal the value of a costly gene therapy
From the article:
"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 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, that the original endpoint was abandoned after yielding mixed results, and that two patients experienced permanent vision loss. Over US$100 million of additional publicly-funded costs are not reflected in the US$850 000 figure."
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