London – AstraZeneca and Ionis’ Wainua (eplontersen) has been approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN.1 Wainua is the only approved medicine for the treatment of ATTRv-PN that can be self-administered via an auto-injector.2-4
The approval by the US Food and Drug Administration (FDA) was based on the positive 35-week interim analysis from the NEURO-TTRansform Phase III trial, which showed patients treated with Wainua demonstrated consistent and sustained benefit on the co-primary endpoints of serum transthyretin (TTR) concentration and neuropathy impairment measured by modified Neuropathy Impairment Score +7 (mNIS+7), and key secondary endpoint of quality of life (QoL) on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN).2,5 Positive results from the NEURO-TTRansform Phase III trial were published in The Journal of the American Medical Association (JAMA) further demonstrating the benefit of Wainua across the spectrum of ATTRv-PN at 35, 66 and 85 weeks.2
Michael J. Polydefkis, M.D., Professor of Neurology at Johns Hopkins University School of Medicine and an investigator in the NEURO-TTRansform study, said: “Many people living with hereditary transthyretin-mediated amyloid polyneuropathy are unable to fully enjoy their lives because of the relentless, progressive and debilitating effects of the disease. 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 debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade.6 Wainua is a ligand-conjugated antisense oligonucleotide (LICA) medicine designed to reduce the production of TTR protein at its source to treat both hereditary and non-hereditary forms of transthyretin-mediated amyloidosis (ATTR).3,7,8
Ruud Dobber, Executive Vice-President, BioPharmaceuticals Business Unit, AstraZeneca, said: “There is an urgent medical need for new therapies for people living with hereditary transthyretin-mediated amyloid polyneuropathy. The US approval of Wainua 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 for people living with this debilitating condition.”
Isabelle Lousada, President and CEO, Amyloidosis Research Consortium, said: “People with hereditary transthyretin-mediated amyloid polyneuropathy, 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. It is exciting to see new innovations coming through and increased efforts to raise awareness in an area that has often been overlooked or neglected.”
As part of a global development and commercialisation agreement, AstraZeneca and Ionis will commercialise Wainua for the treatment of ATTRv-PN in the US and are seeking regulatory approval in Europe and other parts of the world.8 This agreement was recently expanded to include exclusive rights for AstraZeneca to commercialise Wainua in Latin America in addition to all other countries outside the US.9 Wainua was granted Orphan Drug Designation in the US and in the EU for the treatment of ATTR.9,10 Wainua will be available in the US in January 2024.
Brett P. Monia, Ph.D., chief executive officer at Ionis, said: “The FDA approval of Wainua marks an important milestone for people living with hereditary transthyretin-mediated amyloid polyneuropathy, who will now have an effective, well-tolerated treatment that can be self-administered via auto-injector to combat this devastating disease. It is also a pivotal moment for Ionis as Wainua will be the first in a steady cadence of potential commercial launches for the company. We are proud to have discovered and, together with AstraZeneca, developed Wainua, and are grateful to the patients, caregivers and investigators who participated in our clinical studies, as well as for the dedication of our scientists and researchers.”
Eplontersen is currently being evaluated in the CARDIO-TTRansform Phase III trial for treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a systemic, progressive and fatal condition that typically leads to progressive heart failure and often death within three-to-five years from disease onset