The US approval and launch this month is the first in the world for the therapy, a once-monthly antisense treatment that can be self-administered by subcutaneous injection.
According to both companies, Wainua is the only approved medicine for ATTR that can be self-administered without assistance from a healthcare worker.
ATTR is a debilitating disease impacting up to half a million people worldwide that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade.
Wainua is designed to reduce the production of TTR protein in the body to treat both hereditary and non-hereditary forms ATTR.
The approval by the FDA was based on the results from a phase 3 trial, which demonstrated that Wainua reduced levels of TTR by 82%, whereas a placebo group saw an 11% reduction as well as reduced disease progression.
Ruud Dobber, executive vice-President, biopharmaceuticals business unit, 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 for people living with this debilitating condition.”
As part of a global development and commercialisation agreement, AstraZeneca and Ionis will commercialise Wainua for the treatment of ATTR in the US and are seeking regulatory approval in Europe and other parts of the world.
Brett Monia, CEO at Ionis, said the approval marks an ‘important milestone’ for people living with the disease who will now have an ‘effective’ treatment that can be self-administered.
“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,” he added.