FDA accepts NDA for IONIS-TTR-LRx in hereditary transthyretin-mediated amyloid polyneuropathy.- Ionis Pharma

The application has been given a Prescription Drug User Fee Act (PDUFA) action date of 22 December, 2023. In its acceptance letter, the FDA stated that it has not identified any review issues and did not make any additional data requests. The FDA also noted that it is not planning to hold an advisory committee meeting to discuss the application.

The NDA is based on results from the global Phase III NEURO-TTRansform study presented at the International Symposium on Amyloidosis (ISA). In the 35-week interim analysis, eplontersen demonstrated a statistically significant and clinically meaningful change from baseline for its co-primary and key secondary endpoints compared to the external placebo group. In the study, eplontersen achieved a significant mean reduction (p<0.0001) in the co-primary endpoint of serum transthyretin (ttr) concentration compared to baseline. eplontersen also demonstrated a significant treatment effect on the co-primary endpoint of modified neuropathy impairment score +7 (mnis+7), a measure of neuropathic disease progression, with a statistically significant difference in mean change from baseline versus the external placebo group (p><0.0001).></0.0001).></0.0001)>

The study met its key secondary endpoint of change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN), showing that treatment with eplontersen significantly improved patient-reported quality of life compared to the external placebo group (p<0.0001). eplontersen also demonstrated a favorable safety and tolerability profile.></0.0001).>