FDA approves AstraZeneca and Ionis’s Wainua

Betsy Goodfellow | December 22, 2023 | News story | Medical Communications AstraZeneca, FDA, Ionis, Neurology, Wainua 

AstraZeneca and Ionis have announced that the US Food and Drug Administration (FDA) has approved Wainua (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hAATTR-PN or ATTRv-PN) in adult patients.

The drug is the only approved treatment for this indication and can be self-administered with an auto-injector.

The approval follows 35-week data from the NEURO-TTRansform phase 3 trial which demonstrated that patients treated with the drug had a consistent and sustained benefit on the co-primary endpoints of serum transthyretin concentration and neuropathy impairment measured by modified Neuropathy Impairment Score +7, and key secondary endpoint of quality of life.

Michael J Polydefkis MD, professor of Neurology at Johns Hopkins School of Medicine, US, and an investigator on the NEURO-TTRansform study, commented: “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.”

Ruud Dobber, executive vice president, BioPharmaceuticals Business Unit at AstraZeneca, added: “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.”

Betsy Goodfellow

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