AstraZeneca’s Polyneuropathy Treatment Recommended for EU Approval

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Results of a 66-week Phase III trial (NEURO-TTransform) of Wainzua (eplontersen), developed jointly by AstraZeneca and Ionis Pharmaceuticals, led the Committee for Medicinal Products for Human Use (CHMP) of the European Union (EU) to recommend the medicine to the European Commission (EC) for approval in adult patients with hereditary transthyretin-mediated amyloidosis with stage 1 or stage 2 polyneuropathy, (hATTR-PN, or ATTRv-PN) (1).

“This debilitating disease is ultimately fatal if left untreated and can have a significant impact on many aspects of patients’ and caregivers' day-to-day lives,” Laura Obici, MD, a consultant and head of the rare diseases unit at the Amyloidosis Research and Treatment Centre, Istituto Di Ricovero e Cura a Carattere Scientifico Fondazione Policlinico San Matteo, Pavia, Italy, said in a press release from AstraZeneca on Oct. 21, 2024 (1). “Having additional amyloidosis treatment options designed to reduce the production of TTR [transthyretin] protein at its source would potentially give patients more time and ability to do what matters most to them and offer the hope of living longer with a higher quality of life."

In the trial, which has included more than 1400 participants to date, patients treated with Wainzua “demonstrated consistent and sustained benefit” on co-primary endpoints of serum TTR concentration and neuropathy impairment versus placebo, according to the press release (1). If approved by the EC, Wainzua would be the only approved medicine for the treatment of ATTRv-PN in the EU able to be self-administered with an autoinjector on a monthly basis.

“Due to the progressive nature of polyneuropathy of hereditary transthyretin-mediated amyloidosis, it is critical to have timely diagnosis and new therapies to help people have greater control over this potentially fatal disease,” Ruud Dobber, executive vice-president of the biopharmaceuticals business unit of AstraZeneca, said in the release. “Today’s recommendation brings Wainzua one step closer for patients in Europe, and if approved, will offer a new treatment option that can provide consistent TTR suppression and results in improved quality of life.”

An interim analysis of the Phase III trial (at 35 weeks) led FDA to approve Wainzua, under the brand name Wainua, for treatment of ATTRv-PN in adults in December 2023 (2). Similar to what the medicine would signify if CHMP’s recommendation is followed in the EU, FDA’s approval made Wainua the only medicine in the United States that treats ATTRv-PN by self-administration via autoinjector. It was made available in the US beginning in January 2024; at the time, AstraZeneca said it would continue to seek regulatory approvals in Europe and other parts of the world.

Wainzua or Wainua’s generic name, eplontersen, was previously granted orphan drug designation in both the US and EU for treatment of hereditary transthyretin-mediated amyloidosis (1).

References

1. 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.
2. 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.