Ionis Pharmaceuticals and AstraZeneca have obtained approval for Wainua (eplontersen) from the US Food and Drug Administration (FDA) for the treatment of polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis in adults (hATTR-PN or ATTRv-PN).

Wainua is now the only FDA-approved medication for self-administration via an auto-injector in the treatment of ATTRv-PN. As a ligand-conjugated antisense oligonucleotide (LICA), Wainua is specifically designed to decrease the production of the transthyretin (TTR) protein.

The drug will be launched in the US next month. It was given orphan drug designation in the US and in the European Union (EU) for the treatment of ATTR.

AstraZeneca biopharmaceuticals business unit executive vice-president Ruud Dobber 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.”

The approval is driven by the positive 35-week interim analysis from the Phase 3 NEURO-TTRansform study.

The clinical trial revealed that patients treated with Wainua consistently and sustainably demonstrated benefits across the co-primary endpoints, including serum transthyretin (TTR) concentration and neuropathy impairment measured by the modified Neuropathy Impairment Score +7 (mNIS+7).

Additionally, key secondary endpoints related to quality of life (QoL) on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) were also met in the late-stage trial.

Ionis Pharmaceuticals CEO Brett Monia said: “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.”

Eplontersen was discovered by Ionis Pharmaceuticals. In December 2021, the company signed a global development and commercialisation agreement for the ligand-conjugated antisense candidate with AstraZeneca.

As per the agreement, the firms will co-develop and commercialise eplontersen in the US, while AstraZeneca will develop and commercialise it in the rest of the world, with the exception of Latin America.

Eplontersen is presently undergoing assessment in the Phase 3 CARDIO-TTRansform trial for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).