FDA Approves Rare Disease Drug Wainua


Wainua is approved to treat patients with hereditary transthyretin-mediated amyloid polyneuropathy and can be self-administered via an auto-injector.

The FDA has approved Wainua (eplontersen), an antisense medicine to treat patients living with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), a nerve disorder that results in the progressive loss of motor function. These patients accumulate TTR in other major organs, which compromises their function and eventually leads to death within five to 15 years of disease onset.

Developed by Ionis and AstraZeneca, Wainua is designed to reduce the production of transthyretin. It will be available in January 2024 and will be marketed by both companies in the United States. The companies did not release a price, but an AstraZeneca spokesperson said Wainua will be priced competitively and in line with its clinical benefit. Wainua is the only approved medicine to treat ATTRv-PN that can be self-administered via an auto-injector. Patients enrolled in a Medicare plan will access Wainua through Medicare Part D. AstraZeneca’s AZ&Me program will be able to help with out-of-pocket costs.

“Since 2008, the AZ&Me Prescription Savings Program has provided prescription assistance to more than 4 million patients in the United States and Puerto Rico,” the spokesperson said. AstraZeneca also has a program called AstraZeneca Access 360 to help navigate patients’ insurance coverage and direct patients to appropriate sources for financial assistance for which they may be eligible.

The approval is based on the positive 35-week interim analysis from the NEURO-TTRansform phase 3 trial, which showed patients treated with Wainua demonstrated consistent and sustained benefit on the co-primary endpoints of serum transthyretin (TTR) concentration and neuropathy impairment. Wainua achieved an 81.2% reduction in serum transthyretin (TTR) concentration from baseline, demonstrating reduced TTR protein production. Eplontersen demonstrated a favorable safety and tolerability profile.

The trial also demonstrated improvement in the key secondary endpoint of quality of life. Results from an analysis through week 85 were published in The Journal of the American Medical Association in September 2023.

Eplontersen is currently being evaluated in the CARDIO-TTRansform phase 3 trial to treat patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a systemic and progressive condition that typically leads to heart failure and often death within three-to-five years. This study has enrolled 1,400 patients, and results are expected in 2025.

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