An ultra-rare disease drug missed on function but hit on survival and mortality. Is it enough for the FDA?
A California company developing a drug for an ultra-rare, universally fatal neurological disease manifesting in infancy reported Wednesday that its therapy missed the primary endpoint in a Phase II/III study, but showed good enough survival and mortality data to potentially persuade the FDA.
Retrotope revealed its candidate for infantile neuroaxonal dystrophy, or INAD, did not achieve statistical significance in a neurological assessment, showing no differences between patients who received treatment and those in a natural history study. Researchers did, however, see improvement in progression-free survival and a decrease in mortality, both of which mustered statistical significance in secondary endpoints.
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