Wainua

— THERAPEUTIC CATEGORIES —
  • Inborn errors of metabolism
PAGE CONTENTS
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  • Generic Name & Formulations
  • Indications
  • Dosage and Administration
  • Contraindications
  • Boxed Warnings
  • Warnings/Precautions
  • Pharmacokinetics
  • Interactions
  • Adverse Reactions
  • Clinical Trials
  • Note
  • Patient Counseling

    • Wainua Generic Name & Formulations

    Legal Class

    Rx

    General Description

    Eplontersen 45mg/0.8mL; soln for SC inj; preservative-free; contains sodium, phosphorus.

    Pharmacological Class

    Transthyretin-directed antisense oligonucleotide.

    How Supplied

    Single-dose autoinjector—1

    Storage

    Store refrigerated at 2°C to 8°C (36°F to 46°F) in the original carton protected from light.

    If needed, Wainua can be kept at room temperature (up to 30°C [86°F]) in the original carton for up to 6 weeks. If not used within the 6 weeks stored at room temperature, discard Wainua. Do not freeze. Do not expose to heat.

    Manufacturer

    AstraZeneca Pharmaceuticals

    Generic Availability

    NO

    Mechanism of Action

    Eplontersen is an antisense oligonucleotide-GalNAc conjugate that causes degradation of mutant and wild-type TTR mRNA through binding to the TTR mRNA, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.

    Wainua Indications

    Indications

    Polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

    Wainua Dosage and Administration

    Adult

    Give by SC inj into abdomen or upper thigh. For healthcare provider or caregiver: can inject into back of upper arm. 45mg once monthly.

    Children

    Not established.

    Administration

    Prior to initiation, train patients and/or caregivers on proper preparation and administration of Wainua.

    Remove the single-dose autoinjector from the refrigerator 30 minutes prior to the injection and allow to warm to room temperature.

    Inspect visually for particulate matter and discoloration prior to administraiton. Do not use if cloudiness, particulate matter, or discoloration is observed prior to administration.

    Give as a subcutaneous injection into the abdomen or upper thigh region. May use the back of the upper arm as an injection site if a healthcare provider or caregiver administers the injection.

    Wainua Contraindications

    Not Applicable

    Wainua Boxed Warnings

    Not Applicable

    Wainua Warnings/Precautions

    Warnings/Precautions

    Reduced serum Vit. A levels may develop; give supplementation at the recommended daily allowance of Vit. A. Refer to an ophthalmologist if ocular symptoms develop (eg, night blindness, dry eyes). Moderate or severe hepatic impairment, severe renal impairment, or ESRD. Pregnancy. Nursing mothers.

    Pregnancy Considerations

    No available data on the use in pregnant women to inform drug-associated risk of adverse developmental outcomes.

    Wainua decreases serum vitamin A levels, and vitamin A supplementation is advised for patients taking Wainua. Vitamin A is essential for normal embryofetal development.

    There were no adverse developmental effects observed when eplontersen or a mouse-specific surrogate was given to mice prior to mating and continuing throughout organogenesis.

    Nursing Mother Considerations

    Risk Summary

    • Consider the developmental and health benefits of breastfeeding along with the mother's clinical need for Wainua and any potential adverse effects on the breastfed infant from Wainua or from the underlying maternal condition.

    Pediatric Considerations

    Safety and efficacy have not been established.

    Geriatric Considerations

    No overall differences in safety or efficacy between patients ≥65 years of age and younger adults, but greater sensitivity in older individuals cannot be ruled out.

    Renal Impairment Considerations

    Not studied in patients with severe renal impairment or end-stage renal disease.

    Hepatic Impairment Considerations

    Not studied in patients with moderate or severe hepatic impairment.

    Wainua Pharmacokinetics

    Absorption

    Time to maximum plasma concentration: ~2 hours.

    Distribution

    Volume of distribution: 12 L (central); 11,100 L (peripheral). 

    Metabolism

    Hepatic. 

    Elimination

    Half-life: ~3 weeks. 

    Wainua Interactions

    Not Applicable

    Wainua Adverse Reactions

    Adverse Reactions

    Decreased Vit. A, vomiting, proteinuria, inj site reactions (eg, erythema, pain, pruritus), blurred vision, cataract.

    Wainua Clinical Trials

    Clinical Trials

    The approval was based on data from the randomized, open-label, multicenter phase 3 NEURO-TTRansform study (ClinicalTrials.gov Identifier: NCT04136184) which assessed the efficacy and safety of eplontersen in adults with ATTRv-PN. Study participants were randomly assigned 6:1 to receive either eplontersen 45mg subcutaneously once every 4 weeks (n=144) or inotersen 284mg subcutaneously once weekly (n=24). 

    According to 35-week interim analysis, treatment with eplontersen met the efficacy endpoints achieving a statistically significant and clinically meaningful change from baseline in the modified Neuropathy Impairment Score +7 (mNIS+7; a measure of neuropathic disease progression) (mean least squares [LS] treatment difference, -9.0 [95% CI, -13.5, -4.5]; P <.001) and in the Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) total score (mean LS treatment difference, -11.8 [95% CI, -16.8, -6.8]; P <.001), compared with the external placebo arm.

    Additionally, patients who received eplontersen demonstrated similar improvements in mNIS+7 and Norfolk QoL-DN score across subgroups including age, sex, race, region, Val30Met variant status, and disease stage. Consistent and sustained benefits in eplontersen-treated patients were also observed in mNIS+7 and Norfolk QoL-DN score at 66 and 85 weeks from baseline.

    Wainua Note

    Not Applicable

    Wainua Patient Counseling

    Patient Counseling

    Recommended Vitamin A Supplementation

    • Advise patients that Wainua treatment leads to a decrease in vitamin A levels. 
    • Advise patients to take vitamin A supplementation.
    • Advise to contact their health care provider if ocular symptoms suggestive of vitamin A deficiency (eg, night blindness, dry eyes) develop and refer to an ophthalmologist.

    Pregnancy

    • Advise to inform health care provider if they are pregnant or plan to become pregnant during Wainua treatment.

    Cost Savings Program

    Wainua Program Support

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