Advisory Board June 25, 2024
FDA last week approved Sarepta Therapeutics’ gene therapy treatment for use in patients ages four and older with Duchenne muscular dystrophy. However, the decision was made against the recommendation of several agency staff members, who wrote that there was “significant uncertainty regarding the benefits of the treatment.”
How the cell and gene therapy pipeline will change the status quo
FDA expands approval of Sarepta gene therapy
Duchenne muscular dystrophy is a condition that causes progressive muscle weakness and can lead to patients being unable to walk by the time they become teenagers. Currently, there’s no cure for the condition, and treatments, which typically consist of steroid medications and physical therapy, are limited.
Last year, FDA gave accelerated approval to...
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