FDA Approves Wainua (autoinjector) for Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults
The FDA has approved Wainua (autoinjector) for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults. This approval provides a new monthly subcutaneous option for managing this progressive, debilitating condition characterized by nerve damage due to transthyretin amyloid deposits. Wainua is a transthyretin-directed antisense oligonucleotide, offering a targeted approach to address the underlying pathology of hATTR amyloidosis. The approval is based on clinical trial data demonstrating efficacy in improving neuropathy outcomes. Clinicians should note the specific dosing and administration requirements, including patient or caregiver training for proper use of the autoinjector. This addition to the treatment landscape may offer convenience with once-monthly dosing, but its place in therapy relative to other agents will depend on individual patient factors and further real-world evidence.
＋ Clinical Details (Mechanism · Dosing · Trial Data · Warnings)
Wainua is a transthyretin-directed antisense oligonucleotide.
Wainua is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
The recommended dosage of Wainua is 45 mg administered by subcutaneous injection once monthly. Administer into the abdomen or upper thigh region; the back of the upper arm can be used if a healthcare provider or caregiver administers the injection. Remove the single-dose autoinjector from the refrigerator 30 minutes prior to injection and allow to warm to room temperature. Inspect visually for particulate matter and discoloration; the solution should appear colorless to yellow. Do not use if cloudiness, particulate matter, or discoloration is observed. Prior to initiation, train patients and/or caregivers on proper preparation and administration. If a dose is missed, administer as soon as possible and resume dosing at monthly intervals from the date of the most recently administered dose.
The efficacy of Wainua was demonstrated in a randomized, open-label, multicenter clinical trial (Study 1; NCT04136184) in adult patients with polyneuropathy caused by hATTR amyloidosis. 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). Efficacy assessments compared the Wainua arm to an external placebo group (N=60) from another study (NCT01737398). Primary endpoints were change from baseline to Week 35 in modified Neuropathy Impairment Scale+7 (mNIS+7) composite score and Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) total score. The mNIS+7 score range is -22.3 to 346.3, with higher scores indicating greater disease severity. The Norfolk QoL-DN score range is -4 to 136, with higher scores indicating greater impairment. At Week 35, Wainua showed statistically significant improvements compared to placebo: for mNIS+7, baseline mean 79.6 (SD 42.3) with change LS mean 0.2 (SEM 1.9) vs. placebo baseline 74.1 (SD 39.0) with change 9.2 (SEM 1.9), treatment difference -9.0 (95% CI -13.5, -4.5), p<0.001; for Norfolk QoL-DN, baseline mean 43.5 (SD 26.3) with change -3.1 (SEM 2.1) vs. placebo baseline 48.6 (SD 27.0) with change 8.7 (SEM 2.1), treatment difference -11.8 (95% CI -16.8, -6.8), p<0.001.
Not reported in label.
Not reported in label.
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