Wainua Drug Information

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

Recommended Dosage

The recommended dosage of WAINUA is 45 mg administered by subcutaneous injection once monthly. Missed Dose Administer WAINUA as soon as possible after a missed dose. Resume dosing at monthly intervals from the date of the most recently administered dose.

General Administration Instructions

Each autoinjector and prefilled syringe contains a single dose of WAINUA. Prior to administration of the autoinjector or prefilled syringe: • Do not use if WAINUA has been dropped or damaged, appears to be tampered with, or if the expiration date has passed. • Remove WAINUA from the refrigerator 30 minutes prior to the injection to reach room temperature. Do not use other warming methods. • Visually inspect WAINUA before use.

The solution should appear colorless to yellow. Do not use if cloudiness, particulate matter, or discoloration is observed. To deliver a 45 mg dose of WAINUA, administer the entire contents of one autoinjector or prefilled syringe, as a subcutaneous injection in the upper thigh or the abdomen.

The back of the upper arm can also be used as an injection site if a healthcare provider or caregiver administers the injection. Do not inject WAINUA into the area 2 inches (5 cm) around the navel or into areas where the skin is red, warm, tender, bruised, scaly or hard. Rotate the injection site with each injection.

Administration Instructions for WAINUA Autoinjector

The autoinjector is intended for self-administration by patients or administration by caregivers. Prior to initiation, train patients and/or caregivers on proper preparation and administration of WAINUA autoinjector.

Administration Instructions for WAINUA Prefilled Syringe (Healthcare Providers Only) The prefilled syringe must be administered by a healthcare provider. Refer to Figure 1 to identify the prefilled syringe components for use in the administration steps. Do not remove the needle cover until you are ready to inject WAINUA.

Do not touch the needle guard activation clips to prevent premature activation of the needle safety guard. Figure 1: WAINUA Prefilled Syringe Components Administration Steps Step 1 Remove the prefilled syringe from its tray. Hold the syringe body and remove the needle cover by pulling straight off.

Do not recap. If small air bubbles are present, do not expel them prior to administration. Step 2 Choose an injection site (i.e., back of upper arm, upper thigh, or abdomen).

Gently pinch the skin and insert the needle subcutaneously at approximately a 45° angle into the chosen injection site. Step 3 Once the entire dose has been injected, the needle safety device will be triggered. As you let go of the plunger, the needle is automatically pulled from the skin, and into the device; the entire needle will be covered by the needle guard.

Figure_1

Clinical Trials Experience

Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of WAINUA cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. In Study 1, a total of 144 patients with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) were randomized to WAINUA and received at least one dose of WAINUA. The mean duration of treatment was 15 months (range: 1.9 to 19.4 months).

The median patient age at baseline was 52 years and 69% of the patients were male. Fifty-nine percent of patients had the Val30Met variant in the transthyretin gene; the remaining patients had one of 19 other variants. The mean duration from onset of polyneuropathy symptoms was 68 months.

Table 1 lists the adverse reactions that occurred in at least 5% of patients treated with WAINUA in Study 1. Laboratory Tests Vitamin A Decrease In Study 1, patients were instructed to take the recommended daily allowance of vitamin A. All patients treated with WAINUA had normal vitamin A levels at baseline, 95% of patients developed low vitamin A levels during the study.

In some cases, the decreased vitamin A level was reported as an adverse reaction.

Pregnancy Risk Summary There are no available data on WAINUA use in pregnant women to inform drug-associated risk of adverse developmental outcomes. WAINUA treatment leads to a decrease in serum vitamin A levels, and vitamin A supplementation is advised for patients taking WAINUA. Vitamin A is essential for normal embryofetal development; however, excessive levels of vitamin A are associated with adverse developmental effects.

The effect of vitamin A supplementation on the fetus in the setting of a reduction in maternal serum TTR caused by WAINUA administration is unknown. No adverse developmental effects were observed when eplontersen or a mouse-specific surrogate was administered to mice prior to mating and continuing throughout organogenesis. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively.

The background risk of major birth defects and miscarriage for the indicated population is unknown. Data Animal Data Subcutaneous administration of eplontersen mg/kg or a mouse-specific surrogate (25 mg/kg) to male and female mice weekly prior to and during mating and administration continued every other day in females throughout the period of organogenesis resulted in no adverse effects on embryofetal development.

Pediatric Use Safety and effectiveness in pediatric patients have not been established.