The U.S. Food and Drug Administration (FDA) has granted approval to Wainua (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. Eplontersen is the only approved medicine for the treatment of ATTRv-PN that can be self-administered via an auto-injector.
The FDA approval of Eplontersen was based on the positive 35-week interim analysis from the Phase 3 NEURO-TTRansform study. This study demonstrated consistent and sustained benefit in halting neuropathy disease progression and improving neuropathy impairment and quality of life for patients treated with Eplontersen. The co-primary endpoints of serum transthyretin (TTR) concentration and neuropathy impairment measured by modified Neuropathy Impairment Score +7 (mNIS+7) were met, along with the key secondary endpoint of quality of life (QoL) measured by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN).
The positive results from the Phase 3 NEURO-TTRansform study were published in The Journal of the American Medical Association (JAMA), further underscoring the efficacy of Eplontersen in treating ATTRv-PN. These results were consistent across the spectrum of ATTRv-PN at 35, 66, and 85 weeks.
ATTRv-PN is a debilitating disease that leads to peripheral nerve damage and motor disability within five years of diagnosis. Without treatment, it is generally fatal within a decade. Eplontersen, a ligand-conjugated antisense oligonucleotide (LICA) medicine, is designed to reduce the production of TTR protein at its source. By inhibiting the production of misfolded mutated TTR protein, Eplontersen aims to slow down the progression of the disease and improve patients’ quality of life.
The approval of Eplontersen represents a meaningful advancement in the treatment of hereditary transthyretin-mediated amyloid polyneuropathy. Patients living with this condition often face relentless, progressive, and debilitating effects that significantly impact their daily lives. The availability of Eplontersen as a self-administered treatment option provides hope and relief to those managing the disease.
While Eplontersen has demonstrated benefits in treating ATTRv-PN, it is important to be aware of potential safety considerations. Reduced serum vitamin A levels have been observed in patients treated with Eplontersen. It is recommended to supplement with the daily allowance of vitamin A and refer patients to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur. The most common adverse reactions observed in Eplontersen-treated patients were decreased vitamin A levels and vomiting.