Anil Kumar, Retrotope president

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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