WAINUA (eplontersen sodium)

The pharmacokinetic (PK) properties of WAINUA were evaluated following subcutaneous administration of single and multiple doses (once every 4 weeks) in healthy subjects and multiple doses (once every 4 weeks) in patients with hATTR amyloidosis. Eplontersen C max and AUC showed a slightly greater than dose-proportional increase following single subcutaneous doses ranging from 45 to 120 mg (i.e., 1- to 2.7-times the recommended dose) in healthy volunteers. Population estimates (mean ± SD) of stead...

The efficacy of WAINUA was demonstrated in a randomized, open-label, multicenter clinical trial in adult patients with polyneuropathy caused by hATTR amyloidosis (Study 1; NCT04136184). Patients were randomized in a 6:1 ratio to receive either 45 mg of WAINUA once every 4 weeks (N=144), or 284 mg of inotersen once per week (N=24), respectively, as subcutaneous injections. Ninety-seven percent of WAINUA-treated patients and 83% of inotersen-treated patients completed at least 35 weeks of the assigned treatment. Efficacy assessments were based on a comparison of the WAINUA arm of Study 1 with an...

The following clinically significant adverse reactions are discussed in greater detail in other sections of the labeling:

• Reduced Serum Vitamin A Levels and Recommended Supplementation [see Warnings and Precautions (5.1) ]. Most common adverse reactions (that occurred in at least 9% of patients treated with WAINUA) were vitamin A decreased and vomiting. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact AstraZeneca at 1-800-236-9933 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Cli...

None. None. ( 4 )

Reduced Serum Vitamin A Levels and Recommended Supplementation : Supplement with the recommended daily allowance of vitamin A. Refer to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur. ( 5.1 ) 5.1 Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA treatment leads to a decrease in serum vitamin A levels [see Adverse Reactions (6.1) , Use in Specific Populations (8.1) , and Clinical Pharmacology (12.2) ] . Supplementation at the recommended daily a...