Wainua FDA Approval History
Last updated by Judith Stewart, BPharm on Feb 21, 2024.
FDA Approved: Yes (First approved December 21, 2023)
Brand name: Wainua
Generic name: eplontersen
Dosage form: Injection
Company: Ionis Pharmaceuticals and AstraZeneca
Treatment for: Transthyretin-Related Amyloidosis
Wainua (eplontersen) is a transthyretin-directed antisense oligonucleotide indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
- Hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) is a rare, life-threatening disease caused by a mutation in the TTR gene, resulting in the accumulation of misfolded mutated transthyretin (TTR) protein in the peripheral nerves. ATTRv-PN is characterized by debilitating nerve damage throughout the body and the progressive loss of motor functions, such as walking.
- Wainua contains eplontersen which is a ligand-conjugated antisense (LICA) medicine that works to treat ATTRv-PN by reducing the production of TTR protein.
- FDA approval was based on results of the Phase 3 NEURO-TTRansform study which showed patients treated with Wainua demonstrated benefit on the co-primary endpoints of serum TTR concentration and neuropathy impairment, and key secondary endpoint of quality of life.
- Wainua is administered by subcutaneous injection once monthly.
- Warnings and precautions associated with Wainua include reduced serum vitamin A levels. Patients should supplement with the recommended daily allowance of vitamin A.
- Common adverse reactions include decreased vitamin A levels and vomiting.
- Eplontersen is also being studied in the treatment of ATTR cardiomyopathy (ATTR-CM).
Development timeline for Wainua
Further information
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