AstraZeneca’s Wainzua Gains Another Approval in England and Wales

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The National Institute for Health and Care Excellence (NICE) has published a final draft guidance recommending Wainzua (eplontersen) for treatment of hereditary transthyretin-related amyloidosis (ATTR) in adults with stage 1 or 2 polyneuropathy (PN) in England and Wales, AstraZeneca announced in a press release on Oct. 29, 2024 (1).

“We are delighted that eligible patients now have access to this treatment for this significant disease which can be delivered in-clinic or in their own home to support more personalized care,” Tom Keith-Roach, president of AstraZeneca in the United Kingdom, said in the press release (1).

This recommendation of Wainzua closely followed that of the Committee for Medicinal Products for Human Use (CHMP) of the European Union for approval by the European Commission, announced by AstraZeneca on Oct. 21, 2024 and 10 months after FDA approved eplontersen under the brand name Wainua for treatment of adults in the United States in December 2023 (2,3).

"ATTR amyloidosis is a serious, rare disease where people experience neuropathy symptoms, such as pain, discomfort, progressive weakness, and loss of sensation in the legs and arms, and mobility difficulties,” Paul Pozzo, chairman of Amyloidosis UK, said in the Oct. 29 press release. “There is no cure for ATTR amyloidosis, but today’s announcement offers an alternative treatment option to support those living with this condition in the UK ATTR community."

As with CHMP’s recommendation, NICE made its decision based on a 66-week Phase III trial (NEURO-TTRansform) (1).

“Eplontersen provides a new, disease-modifying treatment option, and this is positive news for eligible patients with ATTR-PN and the wider amyloidosis community in the UK,” Marianna Fontana, a professor of cardiology and honorary consultant cardiologist of the National Amyloidosis Centre, Division of Medicine, University College London, said in the press release. “Without treatment, ATTR-PN can lead to extreme polyneuropathy, severe disability, and premature death. In the NEURO-TTRansform Phase III clinical trial, eplontersen significantly lowered serum transthyretin concentration, resulting in less neuropathic impairment and an improved quality of life for patients.”

ATTR-PN, according to AstraZeneca, is caused by a mutation in the transthyretin (TTR) gene responsible for TTR production in the liver (1). The mutation causes the TTR protein to misfold, leading to amyloid fibril accumulation, in turn damaging peripheral nerves in the body. AstraZeneca said ATTR-PN is “frequently under-recognized” because its clinical manifestations are frequently heterogeneous and non-specific (1).

Left untreated, the severe polyneuropathy and motor disability that develops within five years of the onset of ATTR-PN can result in death within a decade (1).

References

1. AstraZeneca. AstraZeneca Secures Accelerated NICE Recommendation for Wainzua (eplontersen) in England and Wales Following MHRA License to Treat Hereditary Transthyretin Amyloidosis in Adult Patients with Stage 1 or 2 Polyneuropathy. Press Release. Oct. 29, 2024.
2. AstraZeneca. Wainzua (eplontersen) Recommended for Approval in the EU by CHMP for the Treatment of Adult Patients with Polyneuropathy Associated with Hereditary Transthyretin-Mediated Amyloidosis. Press Release. Oct. 21, 2024.
3. AstraZeneca. Wainua (eplontersen) Granted First-Ever Regulatory Approval in the US for the Treatment of Adults with Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis. Press Release. Dec. 21, 2023.