AstraZeneca and Ionis Pharmaceuticals have been awarded US Food and Drug Administration (FDA) approval for their jointly developed treatment Wainua (eplontersen) for a treating a rare neurodegenerative disease.

Wainua will be the sole self-administered auto-injector therapy available to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN or ATTRv-PN) in adults. Entering the US market in January 2024, the ligand-conjugated antisense oligonucleotide (ASO) works to slow disease progression by decreasing the production of misfolded transthyretin (TTR) protein which, left unchecked, builds up as amyloid fibrils and causes tissue damage that advances the disease.

The approval was based on 35-week interim data from the Phase III NEURO-TTRansform study (NCT04136184) which showed that patients evaluated at 35 weeks showed “consistent and sustained” benefit in neurologic impairment—as determined by a neuropathy impairment score +7 (mNIS+7)—and serum transthyretin (TTR) concentration. In a presentation at the 2023 American Academy of Neurology held in Boston, Massachusetts in April, AstraZeneca and Ionis shared that the Phase III study achieved its three co-primary endpoints in TTR concentration, neurologic impairment, and quality of life evaluated at week 66. The study also met all of its secondary endpoints.

As a result of the global development and commercialization agreement signed by the two companies in 2021, AstraZeneca holds exclusive rights to commercialise the drug outside the US and is actively working towards gaining regulatory approval in Europe and other countries.

While GlobalData projects Wainua to rake in $1.47 bn by 2029, the drug will enter an exclusive ATTR treatment circle currently dominated by Alnylam Pharmaceuticals. The Cambridge, Massachusetts-based company markets synthetic small interfering RNA (siRNA) drugs Onpattro (patisiran) and Amvuttra (vutrisiran) as treatments for hATTR-PN. Ionis already sells ASO Tegsedi (inotersen) for the indication.

AstraZeneca and Ionis are also looking to challenge Pfizer’s monopoly in the transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) market as Wainua is actively being evaluated as a treatment for ATTR-CM in the Phase III CARDIO-TTRansform study. Pfizer’s Vyndaqel (tafamidis meglumine) is the only FDA-approved therapy for this indication. GlobalData expects Vyndaqel to bring in $4.49 bn in sales by 2026.

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Alnylam originally sought to topple Pfizer’s ATTR-CM indication dominance with a label expansion for Onpattro, but abandoned its plans after the FDA issued a complete response letter citing that the drug had insufficient evidence to demonstrate clinical efficacy in the indication.

GlobalData is the parent company of Pharmaceutical Technology.