Approvals

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, Bio Pharmaceuticals 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.”

Brett P Monia PhD, chief executive officer at Ionis, stated: “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.”