Ionis and AstraZeneca’s WAINZUA gets EU approval for ATTRv-PN treatment

Ionis Pharmaceuticals (IONS) announced that Ionis and AstraZeneca’s (AZN) WAINZUA eplontersen has been approved in the European Union EU for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, commonly referred to as hATTR-PN or ATTRv-PN. WAINZUA is the only approved medicine in the EU for the treatment of ATTRv-PN that can be self-administered monthly via an auto-injector. The approval by the European Commission EC follows the positive opinion of the Committee for Medicinal Products for Human Use CHMP . ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade. WAINZUA is a once-monthly RNA-targeted medicine designed to reduce the production of TTR protein at its source. “Today’s approval of WAINZUA in Europe offers adults with hereditary transthyretin-mediated amyloidosis with polyneuropathy a new, self-administered treatment option that provides consistent suppression of transthyretin production and improves neuropathy impairment and quality of life,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “With approvals in North America, UK and now across the EU, we are proud of the continued progress as we and our partner, AstraZeneca, rapidly and effectively deliver WAINZUA to people around the world.” awarded $ government contract