Feb. 11 at 8:36 AM
ATTR-PN vs ATTR-CM FDA hold update — here’s why the gap in hold removals makes sense:
Patient populations differ — ages, comorbidities, and risk profiles vary.
ATTR-PN is mostly ex-US — high unmet need, no competing
$ALNY drugs.
Trial size matters — ATTR-PN had 47 enrolled (only needed 3 more, now 13), CM is global with 1200+ patients.
Drug/dose consistency — same drugs, same doses, minor safety signal (1 patient with 2” duodenal ulcer, no liver failure) → likely minimal delay.
Best-in-class survival data — Intellia’s Nov update shows strong efficacy; FDA likely won’t drag a long hold with such outcomes, especially with current therapies less effective.
