AstraZeneca (AZ) and Ionis’ Wainua (eplontersen) has been approved by the US Food and Drug Administration (FDA) to treat adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN or ATTRv-PN), a rare disease that leads to peripheral nerve damage with motor disability within five years of diagnosis.
Generally fatal within a decade without treatment, ATTRv-PN affects approximately 40,000 people worldwide.
In both hereditary and non-hereditary forms of transthyretin-mediated amyloidosis, transthyretin (TTR) protein builds up as fibrils in tissues and interferes with their normal functions. As the TTR protein fibrils accumulate, more tissue damage occurs and the disease worsens.
Wainua, which is designed to reduce the production of TTR protein at its source, is now the only approved medicine for ATTRv-PN that can be self-administered via an auto-injector.
The FDA’s decision on the therapy was supported by a positive 35-week interim analysis from the late-stage NEURO-TTRansform trial, in which Wainua was associated with consistent and sustained benefit in the co-primary endpoints of serum TTR concentration and neuropathy impairment, as well as the key secondary endpoint of quality of life.
Positive results from the trial published in the Journal of the American Medical Association further demonstrate the benefit of Wainua across the spectrum of ATTRv-PN at 35, 66 and 85 weeks, the companies added.
Ruud Dobber, executive vice president, biopharmaceuticals business unit, AZ, said: “There is an urgent medical need for new therapies for people living with [ATTRv-PN].
“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.”
As part of a global development and commercialisation agreement, AZ and Ionis will commercialise Wainua for ATTRv-PN in the US and outlined that they are seeking regulatory approval in Europe and other parts of the world.
The drug is also currently being evaluated in a late-stage trial as a treatment for transthyretin-mediated amyloid cardiomyopathy, a systemic, progressive and fatal condition that typically leads to progressive heart failure and often death within three-to-five years from disease onset.
