How long does gene therapy last?
BioMarin looks almost certain to win FDA approval for its hemophilia A gene therapy, but the latest data suggest its effects may wear off over time, clouding the question of just how valuable the one-time treatment might be.
Yesterday, BioMarin presented details on 13 patients followed for up to four years. The seven patients who got the high dose of gene therapy — the one under FDA review — saw a 95% reduction in bleeding episodes sustained over four years, and their need for doses of costly protein replacement therapy fell by about 96%.
However, their levels of Factor VIII, a protein necessary to prevent bleeds, has been on the decline. No one knows whether — or when — the benefits of the gene therapy will wear off. That probably won’t affect the FDA’s decision, expected in August, but it will weigh on the minds of patients and doctors once BioMarin’s treatment reaches the market.
Read more.
Yesterday, BioMarin presented details on 13 patients followed for up to four years. The seven patients who got the high dose of gene therapy — the one under FDA review — saw a 95% reduction in bleeding episodes sustained over four years, and their need for doses of costly protein replacement therapy fell by about 96%.
However, their levels of Factor VIII, a protein necessary to prevent bleeds, has been on the decline. No one knows whether — or when — the benefits of the gene therapy will wear off. That probably won’t affect the FDA’s decision, expected in August, but it will weigh on the minds of patients and doctors once BioMarin’s treatment reaches the market.
Read more.
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