FDA Approves Eplontersen for the Treatment of Adult Patients With ATTRv-PN


The drug is the first medicine to be approved for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis that can be self-administered.

The FDA has approved eplontersen (Wainua; AstraZeneca, Ionis Pharmaceuticals) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) in adults. This approval makes eplontersen the only medicine to be approved for the treatment of ATTRv-PN that can be self-administered with an auto-injector.

“People with hereditary ATTRv-PN, and other forms of amyloidosis, are often misdiagnosed since symptoms can mirror other conditions. The path to getting an accurate diagnosis can often be a long, arduous journey and it is critical that a timely and accurate diagnosis is made not only for the individual experiencing symptoms but for their families and loved ones,” said Isabelle Lousada, president and CEO of Amyloidosis Research Consortium, in a press release.

Eplontersen is a ligand-conjugated antisense oligonucleotide medicine that reduces the production of transthyretin (TRR) protein at its source, treating both hereditary and non-hereditary forms of transthyretin-mediated amyloidosis (ATTR). ATTRv-PN is a disease that leads to peripheral nerve damage with motor disability within 5 years of diagnosis. Without treatment, it can be fatal within a decade. Eplontersen was previously granted an orphan drug designation in the United States and Europe for the treatment of ATTR.

“There is an urgent medical need for new therapies for people living with [ATTRv-PN]. The United States approval of [eplontersen] offers a new treatment option that provides consistent and sustained reduction in serum TTR concentration compared to baseline while halting disease progression and improving quality of life [QoL] for people living with this debilitating condition,” said Ruud Dobber, executive vice president of biopharmaceuticals business unit, AstraZeneca, in the press release.

The approval comes after results from the phase 3 NEURO-TTRansform trial, a 35-week, global, open-label, randomized interim analysis that enrolled adult patients with ATTRv-PN stage 1 or 2 that evaluated the efficacy and safety of eplontersen compared to the placebo, inotersen (Tegsedi; Ionis Pharmaceuticals). The efficacy and safety data comparison of eplontersen vs placebo was based on data up to week 66. Patients were followed on treatment until week 85, at which time they were given the option to transition to an open-label extension study.

The study results demonstrated that patients treated with eplontersen had experienced a consistent and sustained benefit on the co-primary endpoints of serum TRR concentration and neuropathy impairment. The secondary endpoint was QoL. Further, the study results indicate a benefit of eplontersen across the spectrum of ATTRv-PN at 35, 66, and 85 weeks. The most common adverse effects reported by patients treated with eplontersen (≥9%) were a decrease in serum vitamin A levels (15%) and vomiting (9%).

“Many people living with hereditary ATTRv-PN are unable to fully enjoy their lives because of the relentless, progressive and debilitating effects of the disease. [The] approval of [eplontersen] represents a meaningful advancement in treatment, one that gives those who are living with ATTRv-PN help managing the disease,” said study investigator Michael J. Polydefkis, MD, professor of neurology at Johns Hopkins University School of Medicine, in the press release.


Businesswire. WAINUA™ (eplontersen) Granted First-Ever Regulatory Approval in the US for the Treatment of Adults With Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis.News release. December 21, 2023. Accessed December 22, 2023. https://www.businesswire.com/news/home/20231221289590/en

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