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A New Horizon in Rare Disease Treatment: FDA Approves Wainua for ATTR-PN


23 December 2023

The FDA has approved Wainua, an innovative treatment for hereditary transthyretin-mediated amyloidosis polyneuropathy (ATTR-PN), developed by AstraZeneca and Ionis Pharmaceuticals. This novel drug is the only approved medicine for the treatment of ATTRv-PN and offers hope to patients with this rare and debilitating disease.

AstraZeneca and Ionis Pharmaceuticals have announced that the US Food and Drug Administration (FDA) approved Wainua (eplontersen), marking a landmark decision in the treatment of rare diseases. This groundbreaking drug is for the treatment of hereditary transthyretin-mediated amyloidosis polyneuropathy (ATTR-PN). Wainua demonstrated significant benefits in clinical trials, heralding a new era of hope for patients suffering from this life-altering condition.

ATTR-PN, a debilitating disease, leads to severe peripheral nerve damage and motor disabilities and can be fatal within a decade of diagnosis if left untreated. The pathogenesis involves misfolded transthyretin (TTR) protein accumulating as amyloid fibrils in various body tissues, progressively impairing their function. Globally, it affects approximately 40,000 patients.

Dr. Michael Polydefkis, Professor of Neurology at Johns Hopkins University and an investigator in study, emphasizes, “Many people living with ATTR-PN are unable to fully enjoy their lives because of relentless, progressive and debilitating effects of 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.”

Wainua, a ligand-conjugated antisense oligonucleotide (LICA), is designed to reduce the production of TTR protein at its source, treating both hereditary and non-hereditary forms of transthyretin-mediated amyloidosis. Its approval is based on the results of the NEURO-TTRansform Phase III trial, which demonstrated consistent and sustained benefits in improving neuropathy impairment and quality of life. Notably, patients experienced significant reductions in serum TTR concentration, along with improvements in neuropathy impairment and quality of life.

Ruud Dobber, Executive Vice-President, BioPharmaceuticals, 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.”

The commercialization of Wainua is a part of a global development agreement between AstraZeneca and Ionis, aiming for regulatory approval in Europe and other regions. The drug's Orphan Drug Designation in the US and EU for ATTR treatment further signifies its importance in rare disease therapy.

Ionis CEO Brett Monia's statement reflects significance of this approval, as Wainua becomes a pivotal solution for people living with hereditary transthyretin-mediated amyloid polyneuropathy. Additionally, the drug is currently being evaluated for treating transthyretin-mediated amyloid cardiomyopathy, a condition leading to progressive heart failure and often death.

The FDA's approval of Wainua marks a significant milestone in rare disease treatment, offering a new, effective, and patient-friendly treatment option for those battling ATTR-PN. 

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