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FDA approved Wainua (autoinjector) for Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in AdultsFDA approved new monthly injection called Wainua for a rare nerve disease in adults

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Key Takeaway
Consider Wainua for monthly subcutaneous treatment of hATTR amyloidosis polyneuropathy 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)
Mechanism of Action

Wainua is a transthyretin-directed antisense oligonucleotide.

Indication & Patient Population

Wainua is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

Dosing & Administration

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.

Key Clinical Trial Data

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.

Warnings & Contraindications

Not reported in label.

Place in Therapy

Not reported in label.

The U.S. Food and Drug Administration (FDA) has approved a new medication called Wainua. It is a monthly injection given just under the skin. It is approved to treat adults with a rare, inherited disease called hereditary transthyretin-mediated amyloidosis, or hATTR amyloidosis. This disease causes harmful protein deposits to build up in the body, which damages nerves and leads to symptoms like numbness, pain, and weakness. Wainua is designed to target the root cause of this disease by reducing the production of the problematic protein.

This approval is for adults living with this specific nerve condition. It provides a new option for managing the disease. The treatment is given once a month using a special auto-injector device, which may offer convenience compared to some other treatments. The FDA's decision was based on clinical trial data showing the drug helped improve nerve-related outcomes for patients.

It's important to understand that this is a new addition to the available treatments. Like all medications, it has specific dosing and administration requirements. Patients or their caregivers will need proper training on how to use the auto-injector correctly. The place of this drug in a patient's overall care plan will depend on many individual factors.

One honest point to remember is that this approval means the drug is now available as an option, but it does not automatically make it the right choice for every person with this condition. The most important step for anyone interested in this treatment is to have a detailed conversation with their own healthcare team. Doctors can help explain if Wainua might be suitable based on a person's unique health situation.

What this means for you:
A new monthly injection is now an option for adults with a specific rare nerve disease.

Study Details

Study typeFda approval
PublishedDec 2023
View Original Abstract ↓
1 INDICATIONS AND USAGE WAINUA is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. WAINUA is a transthyretin-directed antisense oligonucleotide indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. ( 1 )
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