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Wainua Gets First US Approval for Hereditary Transthyretin-Mediated Amyloidosis

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The recent approval of Wainua (eplontersen) by the US Food and Drug Administration (FDA) for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) signifies a significant breakthrough in addressing a debilitating condition. This approval comes following the positive outcomes observed in the NEURO-TTRansform Phase III trial, where Wainua demonstrated consistent and sustained benefits in improving neuropathy impairment and quality of life in patients with ATTRv-PN.

ATTRv-PN, also known as hereditary transthyretin-mediated amyloid polyneuropathy, is a distressing disease characterized by peripheral nerve damage and motor disability, often resulting in significant impairment within five years of diagnosis and a fatal prognosis within a decade without proper treatment. The availability of Wainua as a self-administered medicine through an auto-injector offers new hope for individuals battling this condition.

Wainua Shows Consistent Benefit in Amyloidosis Parameters

The pivotal NEURO-TTRansform Phase III trial, which included a cohort of 1,074 participants, demonstrated promising outcomes for Wainua. The trial’s interim analysis indicated substantial and sustained benefits in critical parameters such as serum transthyretin (TTR) concentration, neuropathy impairment measured by modified Neuropathy Impairment Score +7 (mNIS+7), and quality of life (QoL) measured by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN). These positive results were further validated by their publication in The Journal of the American Medical Association (JAMA) at intervals of 35, 66, and 85 weeks, showcasing the consistent benefit of Wainua across the spectrum of ATTRv-PN.

Dr. Michael J. Polydefkis, Professor of Neurology at Johns Hopkins University School of Medicine and an investigator in the NEURO-TTRansform study, emphasized the significance of this approval. He highlighted the challenges faced by individuals with hereditary transthyretin-mediated amyloid polyneuropathy, hindering their ability to lead fulfilling lives due to the disease’s relentless and debilitating effects. The approval of Wainua represents a meaningful advancement that provides a ray of hope for managing this condition and improving the lives of those affected.

Amyloidosis

 

ATTRv-PN is a condition that often leads to misdiagnosis due to symptoms that may mimic other ailments, making the path to an accurate diagnosis arduous and prolonged. Isabelle Lousada, President and CEO of the Amyloidosis Research Consortium, emphasized the importance of timely and accurate diagnosis not only for individuals experiencing symptoms but also for their families. The arrival of Wainua represents a promising step forward in an area of medicine that has often been overlooked or inadequately addressed.

The regulatory approval of Wainua in the US also marks the commencement of efforts to seek approvals in other parts of the world. AstraZeneca and Ionis, the developers of Wainua, are pursuing regulatory reviews in Europe and other regions as part of a global development and commercialization agreement. This international expansion aims to make Wainua available to a broader population, addressing the unmet medical needs of patients worldwide. The drug’s availability in the US from January 2024 signifies an impending milestone in the fight against ATTRv-PN.

Beyond ATTRv-PN to Addressing Transthyretin-Mediated Amyloid Cardiomyopathy

Moreover, the potential of eplontersen (Wainua) is not limited to ATTRv-PN. Ongoing investigations are evaluating its efficacy in treating transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), another severe and life-threatening condition. This exploration into different forms of amyloidosis underscores the dedication of researchers and organizations to finding solutions for these complex and debilitating diseases.

In conclusion, the FDA approval of Wainua represents a pivotal moment in the journey toward effective treatments for hereditary transthyretin-mediated amyloidosis. It signifies a beacon of hope for patients, caregivers, and the medical community, offering much-needed therapy and paving the way for further advancements in addressing various forms of amyloidosis.

 

Resource: AstraZeneca, December 22, 2023

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