FDA approves Wainua for polyneuropathy of hereditary transthyretin-mediated amyloidosis

Key Takeaways

• Wainua has received regulatory approval for polyneuropathy of hereditary transthyretin-mediated amyloidosis.
• Wainua will be available in the U.S. in January 2024.

The FDA has granted regulatory approval for Wainua for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, according to a press release.

Wainua (eplontersen; Ionis, Astrazeneca) is currently the only approved self-administered treatment for polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTER-PN or ATTERv-PN), the company said.

According to the press release, positive results from the 35-week interim analysis of the phase three NEURO-TTRansform study led to the approval in the United States. The analysis demonstrated consistent and sustained benefits for serum transthyretin concentration, neuropathy impairment and quality of life among patients receiving Wainua.

"Many people living with hereditary transthyretin-mediated amyloid polyneuropathy are unable to fully enjoy their lives because of the relentless, progressive and debilitating effects of the disease," Michael J. Polydefkis, MD, professor of neurology at Johns Hopkins University School of Medicine and an investigator in the NEURO-TTRansform study, said in the release. "Approval of Wainua represents a meaningful advancement in treatment, one that gives those who are living with transthyretin-mediated amyloid polyneuropathy help managing the disease."

Regulatory approval for the drug is underway in Europe, as well as other parts of the world, according to the release.

Wainua will be available in the U.S. in January 2024.