Key Takeaways
- AstraZeneca’s Wainzua (eplontersen) recommended for EU approval for treating hereditary transthyretin-mediated amyloidosis (ATTRv-PN) with polyneuropathy.
- NEURO-TTRansform trial shows Wainzua improves neuropathy impairment and quality of life.
- If approved, Wainzua will be the first EU-approved, monthly self-administered therapy for ATTRv-PN.
AstraZeneca and Ionis Pharmaceuticals have announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has recommended the approval of Wainzua (eplontersen) for adult patients with hereditary transthyretin-mediated amyloidosis (ATTRv-PN), specifically targeting stages 1 and 2 polyneuropathy.
ATTRv-PN, a rare genetic condition, causes progressive nerve damage and is potentially fatal within a decade if untreated. Wainzua, an RNA-targeted therapeutic, directly suppresses production of the misfolded transthyretin (TTR) protein, addressing the disease at its source. If the European Commission grants approval, Wainzua would become the only EU-approved therapy for ATTRv-PN that can be administered at home via monthly self-injection, offering a new level of independence to patients who face debilitating symptoms.
The CHMP’s positive opinion is based on findings from the NEURO-TTRansform Phase III trial, which demonstrated that Wainzua provided a consistent reduction in serum TTR concentration and improved neuropathy symptoms. Across 66 weeks, the trial results showed statistically significant benefits on co-primary endpoints, including the Modified Neuropathy Impairment Score (mNIS+7) and quality of life scores, versus placebo.
Additionally, Wainzua was well-tolerated, maintaining a favorable safety profile throughout the study. Dr. Laura Obici, Head of the Rare Diseases Unit at the Amyloidosis Research and Treatment Centre in Pavia, Italy, emphasized the importance of this advancement, stating, “ATTRv-PN profoundly impacts patients and caregivers, affecting daily life and longevity. An effective, easy-to-administer therapy like Wainzua offers these patients hope for an improved quality of life.”
Eplontersen’s Potential to Address Key Needs in ATTRv-PN Treatment
ATTRv-PN, a genetic form of transthyretin amyloidosis, stems from the misfolding of TTR proteins produced by the liver, causing amyloid deposits that damage the nerves and can affect other organs, including the heart and kidneys. Without intervention, patients face severe motor impairments and reduced life expectancy. Current therapies are limited and often require regular healthcare visits for administration. Wainzua’s RNA-targeting mechanism suppresses the production of the faulty TTR protein, thereby slowing disease progression and potentially preventing further nerve damage.
The NEURO-TTRansform study provided critical insights into Wainzua’s effectiveness, including marked improvements in neuropathy scores and overall quality of life measures, as evaluated through the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN). This consistent benefit profile makes Wainzua an appealing treatment option for a population with limited choices.
Ruud Dobber, Executive Vice-President of AstraZeneca’s BioPharmaceuticals Business Unit, highlighted the therapy’s significance, noting that “the progressive nature of ATTRv-PN necessitates prompt diagnosis and treatment to improve patient outcomes. Wainzua, as a monthly self-administered therapy, addresses these needs and offers patients an opportunity for greater independence and quality of life.”
Future Prospects and AstraZeneca’s Role in Cardiovascular and Metabolic Health
AstraZeneca and Ionis are advancing Wainzua’s global reach, with approval already granted in the United States under the name Wainua. AstraZeneca is also conducting further trials, including the ongoing CARDIO-TTRansform Phase III study, which is evaluating eplontersen in treating transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), the largest study of its kind.
ATTR-CM affects the heart and can lead to severe cardiac conditions. With over 1,400 participants enrolled, this study aims to establish eplontersen’s potential to manage ATTR-CM, expanding its application in the treatment of amyloidosis. The development of Wainzua aligns with AstraZeneca’s broader Cardiovascular, Renal, and Metabolism (CVRM) strategy, focusing on organ protection and disease progression control across interconnected health domains.
This initiative reflects AstraZeneca’s commitment to transformative therapies that improve patient outcomes, particularly in conditions with high unmet needs like ATTRv-PN. AstraZeneca continues to invest in its biopharmaceutical pipeline, targeting advancements in organ-protective therapies that may ultimately contribute to longer, healthier lives for patients worldwide.
Resource: Astrazeneca, October 21, 2024
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