RARE Daily

FDA Approves AstraZeneca and Ionis Wainua to Treat hATTR-PN

December 22, 2023

Rare Daily Staff

The U.S. Food and Drug Administration approved AstraZeneca and Ionis’ Wainua for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN.

Wainua is the only approved medicine for the treatment of ATTRv-PN that can be self-administered via an auto-injector.

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. ATTR-PN polyneuropathy is a progressive systemic disease caused by aging or genetic mutations, resulting in misfolded TTR protein and accumulation as amyloid fibrils in the peripheral nerves. TTR protein builds up as fibrils in tissues, such as the peripheral nerves and heart, gastrointestinal system, eyes, kidneys, central nervous system, thyroid and bone marrow. The presence of TTR fibrils interferes with the normal functions of these tissues. As the TTR protein fibrils accumulate, more tissue damage occurs and the disease worsens, resulting in poor quality of life and eventually death. Worldwide, there are an estimated 40,000 patients with ATTRv-PN.

Wainua (eplontersen) 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).

It is currently being evaluated in the CARDIO-TTRansform phase 3 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.

FDA approval was based on the positive 35-week interim analysis from the NEURO-TTRansform phase 3 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). Positive results from the NEURO-TTRansform phase 3 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.

“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,” said Michael Polydefkis, professor of neurology at Johns Hopkins University School of Medicine and an investigator in the NEURO-TTRansform study. “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.”

As part of a global development and commercialization agreement, AstraZeneca and Ionis will commercialize Wainua for the treatment of ATTRv-PN in the United States and are seeking regulatory approval in Europe and other parts of the world. This agreement was recently expanded to include exclusive rights for AstraZeneca to commercialize Wainua in Latin America in addition to all other countries outside the United States Wainua was granted Orphan Drug designation in the United States and in the European Union for the treatment of ATTR. Wainua will be available in the United States in January 2024.

“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.

Photo: Brett Monia, CEO at Ionis

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