In a major development for hereditary transthyretin-mediated amyloidosis (ATTRv-PN or hATTR-PN) patients, the US Food and Drug Administration (FDA) has approved Wainua (eplontersen), developed by AstraZeneca PLC and Ionis Pharmaceuticals Inc. This approval is particularly noteworthy as Wainua is the first and only FDA-approved medication for ATTRv-PN that can be self-administered using an auto-injector.

The decision was based on the 35-week interim analysis of the NEURO-TTRansform Phase III trial. This study highlighted Wainua’s efficacy in consistently and significantly reducing serum transthyretin (TTR) levels and neuropathy impairment. Additionally, it demonstrated an improvement in the quality of life for patients, as measured by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN). These positive results, also published in The Journal of the American Medical Association (JAMA), solidify Wainua’s role in the ATTRv-PN treatment spectrum.

Professor Michael J. Polydefkis, M.D., from Johns Hopkins University School of Medicine and an investigator in the NEURO-TTRansform study, emphasized the impact of Wainua’s approval, stating, “Approval of Wainua represents a meaningful advancement in treatment, one that gives those who are living with transthyretin-mediated amyloid polyneuropathy help managing the disease.”

ATTRv-PN is a rapidly progressing and debilitating disease, leading to peripheral nerve damage and motor disability within five years of diagnosis. Wainua, a ligand-conjugated antisense oligonucleotide (LICA) medicine, is designed to reduce TTR protein production, thereby treating both hereditary and non-hereditary forms of transthyretin-mediated amyloidosis.

Ruud Dobber, Executive Vice-President of AstraZeneca’s BioPharmaceuticals Business Unit, highlighted the urgent need for new therapies in this area, noting Wainua’s capacity to provide consistent TTR concentration reduction and halt disease progression.

Isabelle Lousada, President and CEO of the Amyloidosis Research Consortium, pointed out the challenges in diagnosing ATTRv-PN, as its symptoms often mimic other conditions. She stressed the importance of timely and accurate diagnoses and expressed enthusiasm for the new treatments and awareness efforts in this often-overlooked field.

Under a global development and commercialization agreement, AstraZeneca and Ionis will market Wainua in the US and are seeking regulatory approval in Europe and other regions. The agreement also grants AstraZeneca exclusive rights to commercialize Wainua in Latin America and other countries outside the US. Wainua, which received Orphan Drug Designation in the US and EU, is expected to be available in the US in January 2024.

Brett P. Monia, CEO of Ionis, remarked on the significance of the FDA approval, stating, “The FDA approval of Wainua marks an important milestone for people living with hereditary transthyretin-mediated amyloid polyneuropathy…”

Wainua is also under evaluation in the CARDIO-TTRansform Phase III trial for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a condition usually resulting progressive heart failure and death within three to five years from onset.

  AstraZeneca, ATTRv-PN, Eplontersen, hereditary transthyretin-mediated amyloidosis, Ionis Pharmaceuticals, Isabelle Lousada, Ruud Dobber, US FDA, Wainua