The US approval is the first in the world for Wainua (eplontersen), a once-monthly antisense treatment that can be self-administered by subcutaneous injection. According to AZ and Ionis, it is the only drug for ATTR polyneuropathy that doesn’t need to be administered by a healthcare professional.
ATTR is a debilitating disease affecting 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 works by reducing levels in the body of TTR protein, which builds up as fibrils in tissues and damages them.
