WAINZUA (eplontersen) approved in the EU for the treatment of hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy
Ionis Pharmaceuticals (IONS) announced the European Union approval of WAINZUA (eplontersen) for treating hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or 2 polyneuropathy (ATTRv-PN). The approval follows successful NEURO-TTRansform Phase 3 trial results through 66 weeks.
WAINZUA, marketed as WAINUA in the U.S., is the EU's only approved self-administered monthly auto-injector treatment for ATTRv-PN. The drug demonstrated consistent benefits in serum transthyretin concentration, neuropathy impairment, and quality of life versus placebo, while maintaining a favorable safety profile.
This marks WAINZUA's second major approval, following previous authorizations in North America and the UK. The treatment is being jointly developed and commercialized by Ionis and AstraZeneca in the U.S., with AstraZeneca holding exclusive rights for other global markets.
Ionis Pharmaceuticals (IONS) ha annunciato l'approvazione da parte dell'Unione Europea di WAINZUA (eplontersen) per il trattamento dell'amiloidosi ereditaria mediata dalla transtirettina in pazienti adulti con polineuropatia di stadio 1 o 2 (ATTRv-PN). L'approvazione segue i risultati positivi del NEURO-TTRansform Phase 3 trial dopo 66 settimane.
WAINZUA, commercializzato come WAINUA negli Stati Uniti, è l'unico trattamento approvato nell'UE che può essere somministrato mensilmente tramite auto-iniettore. Il farmaco ha dimostrato benefici costanti nella concentrazione di transtirettina sierica, nel miglioramento della neuropatia e nella qualità della vita rispetto al placebo, mantenendo un profilo di sicurezza favorevole.
Questo segna il secondo importante approvazione per WAINZUA, dopo le precedenti autorizzazioni in Nord America e nel Regno Unito. Il trattamento è sviluppato e commercializzato congiuntamente da Ionis e AstraZeneca negli Stati Uniti, con AstraZeneca che detiene i diritti esclusivi per altri mercati globali.
Ionis Pharmaceuticals (IONS) anunció la aprobación de la Unión Europea para WAINZUA (eplontersen) para el tratamiento de la amiloidosis hereditaria mediada por transtiretina en pacientes adultos con polineuropatía en etapa 1 o 2 (ATTRv-PN). La aprobación sigue los resultados exitosos del NEURO-TTRansform Phase 3 trial a lo largo de 66 semanas.
WAINZUA, comercializado como WAINUA en EE. UU., es el único tratamiento aprobado en la UE que se puede autoadministrar mensualmente mediante un auto-inyector. El fármaco mostró beneficios consistentes en la concentración de transtiretina en suero, en la discapacidad neuropática y en la calidad de vida en comparación con el placebo, manteniendo un perfil de seguridad favorable.
Esto marca la segunda gran aprobación de WAINZUA, después de las autorizaciones previas en América del Norte y el Reino Unido. El tratamiento se desarrolla y comercializa conjuntamente por Ionis y AstraZeneca en EE. UU., con AstraZeneca teniendo derechos exclusivos para otros mercados globales.
아이오니스 제약 (IONS)은 WAINZUA (에플론테르센)이 성인 환자의 유전성 트랜스티레틴 매개 아밀로이드증(ATTRv-PN) 치료를 위한 유럽연합의 승인을 받았다고 발표했습니다. 이 승인은 66주 동안의 NEURO-TTRansform 3상 시험 결과를 따릅니다.
WAINZUA는 미국에서 WAINUA라는 이름으로 판매되며, ATTRv-PN에 대한 유일한 자가 주사 월간 치료제로 유럽연합에서 승인되었습니다. 이 약물은 위약에 비해 혈청 트랜스티레틴 농도, 신경병증 손상 및 삶의 질에서 일관된 이점을 보여주었으며, 안전성 프로필도 긍정적이었습니다.
이는 WAINZUA의 두 번째 주요 승인으로, 북미와 영국에서의 이전 승인에 이어지는 것입니다. 이 치료는 아이오니스와 아스트라제네카가 미국에서 공동 개발 및 상용화하고 있으며, 아스트라제네카는 다른 글로벌 시장에 대한 독점 권리를 보유하고 있습니다.
Ionis Pharmaceuticals (IONS) a annoncé l'approbation par l'Union Européenne de WAINZUA (eplontersen) pour le traitement de l'amylose héréditaire médiée par la transthyretine chez les patients adultes présentant une polynévropathie au stade 1 ou 2 (ATTRv-PN). Cette approbation fait suite aux résultats positifs de l'essai NEURO-TTRansform Phase 3 sur 66 semaines.
WAINZUA, commercialisé sous le nom de WAINUA aux États-Unis, est le seul traitement approuvé dans l'UE qui peut être administré mensuellement via un auto-injecteur. Le médicament a montré des avantages constants en termes de concentration de transthyretine sérique, d'atteinte neuropathique et de qualité de vie par rapport au placebo, tout en maintenant un profil de sécurité favorable.
Ceci marque la deuxième grande approbation de WAINZUA, après les autorisations précédentes en Amérique du Nord et au Royaume-Uni. Le traitement est développé et commercialisé conjointement par Ionis et AstraZeneca aux États-Unis, AstraZeneca détenant les droits exclusifs pour d'autres marchés mondiaux.
Ionis Pharmaceuticals (IONS) hat die Genehmigung der Europäischen Union für WAINZUA (eplontersen) zur Behandlung der hereditären transthyretin-vermittelten Amyloidose bei erwachsenen Patienten mit Polyneuropathie im Stadium 1 oder 2 (ATTRv-PN) bekannt gegeben. Die Genehmigung folgt auf erfolgreiche Ergebnisse der NEURO-TTRansform Phase 3-Studie über 66 Wochen.
WAINZUA, in den USA als WAINUA vermarktet, ist die einzige genehmigte selbstverabreichte monatliche Auto-Injektor-Behandlung für ATTRv-PN in der EU. Das Medikament zeigte konsistente Vorteile in der Serum-Transthyretin-Konzentration, der Beeinträchtigung der Neuropathie und der Lebensqualität im Vergleich zu Placebo und wies ein günstiges Sicherheitsprofil auf.
Dies markiert die zweite große Genehmigung für WAINZUA, nach vorherigen Zulassungen in Nordamerika und dem Vereinigten Königreich. Die Behandlung wird gemeinsam von Ionis und AstraZeneca in den USA entwickelt und vermarktet, wobei AstraZeneca die exklusiven Rechte für andere globale Märkte hält.
- EU approval expands market access for WAINZUA, following US and UK approvals
- Only EU-approved self-administered monthly treatment option for ATTRv-PN
- Strong Phase 3 trial results showing sustained efficacy
- Strategic partnership with AstraZeneca for global commercialization
- None.
Insights
The EU approval of WAINZUA (eplontersen) represents a significant commercial milestone for Ionis Pharmaceuticals and its partner AstraZeneca. This marks the second major regulatory approval following the December 2023 US approval (under brand name WAINUA), creating a multi-region commercial foundation for this rare disease treatment.
The approval addresses hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN), a life-threatening condition with treatment options. Notably, WAINZUA offers a competitive advantage as the only EU-approved ATTRv-PN treatment with monthly self-administration via auto-injector—a meaningful convenience factor for chronic therapy.
Supporting clinical data demonstrated consistent efficacy across multiple endpoints through 66 weeks, including TTR reduction, neuropathy improvement (mNIS+7), and quality of life metrics. This robust efficacy profile coupled with favorable safety positions WAINZUA well in the European market.
With approvals now spanning North America, the UK, and EU, Ionis and AstraZeneca are building substantial global market access. The strategic partnership leverages AstraZeneca's commercial infrastructure for international markets while Ionis maintains co-commercialization rights in the US. Additional regulatory submissions worldwide should further expand the drug's commercial potential in treating this fatal disease with high unmet need.
The EU approval of WAINZUA for ATTRv-PN represents a meaningful therapeutic advancement for patients with this devastating condition. ATTRv-PN progressively destroys peripheral nerves, causing motor disability within five years and death within approximately a decade if untreated.
WAINZUA's mechanism—reducing TTR protein production at its source through RNA targeting—addresses the fundamental pathophysiology driving disease progression. The NEURO-TTRansform Phase 3 results demonstrated both sustained TTR suppression and clinically meaningful improvements in neuropathy impairment, suggesting potential to alter disease trajectory.
The monthly self-administration option is particularly valuable for ATTRv-PN patients, whose progressive neurological impairment makes frequent hospital visits increasingly challenging. This convenience factor likely enhances treatment adherence, maximizing real-world effectiveness.
From a patient-centric perspective, having consistent efficacy through 66 weeks on both objective measures (mNIS+7) and subjective quality-of-life assessments indicates comprehensive symptom management. Given the therapeutic landscape for ATTRv-PN in Europe, WAINZUA fulfills a critical treatment gap for a patient population with historically poor prognosis and helps address the significant burden of care on patients, families, and healthcare systems.
– Second major approval for WAINZUA, which is marketed in the
– EU approval based on NEURO-TTRansform Phase 3 results showing WAINZUA demonstrated consistent and sustained benefit improving neuropathy impairment and quality of life versus placebo –
The approval by the European Commission (EC) follows the positive opinion of the Committee for Medicinal Products for Human Use (CHMP). The approval is based on the positive NEURO-TTRansform Phase 3 trial which showed that through 66 weeks, patients treated with WAINZUA demonstrated consistent and sustained benefit on the co-primary endpoints of serum transthyretin (TTR) concentration and neuropathy impairment measured by modified Neuropathy Impairment Score +7 (mNIS+7), and key secondary endpoint of quality of life (QoL) on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) versus external placebo. WAINZUA continued to demonstrate a favorable safety and tolerability profile throughout the NEURO-TTRansform trial.
ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade. WAINZUA is a once-monthly RNA-targeted medicine designed to reduce the production of TTR protein at its source.
“Today’s approval of WAINZUA in
WAINZUA was approved under the brand name WAINUA for the treatment of ATTRv-PN in the
About TTR Amyloidosis
ATTR is caused by the accumulation of liver-derived misfolded TTR protein in tissues, such as the heart and the peripheral nerves, causing organ damage and failure. ATTR then causes complications, leading to cardiovascular, neurological and renal diseases such as heart failure and chronic kidney disease. There are both hereditary (ATTRv) and non-hereditary (wild-type) forms of ATTR. ATTR is a rapidly progressive and fatal disease that requires timely recognition of symptoms. ATTR has several phenotypes including ATTR-cardiomyopathy (CM), which predominantly impacts the heart, potentially leading to heart failure, ATTR-polyneuropathy (PN), which predominantly affects the peripheral nervous system and mixed phenotype, where patients experience symptoms of both. Worldwide, there are an estimated 300,000 – 500,000 patients with ATTR-CM and about 10,000 – 40,000 patients with ATTRv-PN.
About NEURO-TTRansform
NEURO-TTRansform is a global, open-label, randomized trial evaluating the efficacy and safety of eplontersen in patients with ATTRv-PN at week 35, week 66 and week 85. The final analysis comparing eplontersen to an external placebo group was completed at week 66. All patients were then followed on treatment until week 85 and evaluated four weeks after the last dose in an end-of-trial assessment. Following treatment and the end-of-trial assessments, patients were eligible to enter an open-label extension study to continue receiving eplontersen once every four weeks or enter a 20-week post-treatment evaluation period, which is still ongoing. Full results from the NEURO-TTRansform trial were published in The Journal of the American Medical Association (JAMA) demonstrating the benefit of eplontersen across the spectrum of ATTRv-PN at 35, 66 and 85 weeks of treatment.
About WAINZUA / WAINUA™ (eplontersen)
Eplontersen is a once-monthly RNA-targeted medicine that provides upstream suppression of transthyretin (TTR) production and is designed to precisely target and reduce the production of TTR protein at its source in the liver.
WAINZUA has been approved in the
Eplontersen is also currently being evaluated in the global CARDIO-TTRansform Phase 3 study for the treatment of adults with ATTR-CM. The trial is fully enrolled with more than 1,400 patients – making it the largest, most comprehensive study to date in this patient population. More information on the CARDIO-TTRansform study (NCT04136171) is available at www.clinicaltrials.gov.
WAINUA injection for subcutaneous use 45 mg is indicated for treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
IMPORTANT SAFETY INFORMATION for WAINUA™ (eplontersen)
WARNINGS AND PRECAUTIONS
Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.
ADVERSE REACTIONS
Most common adverse reactions (≥
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About Ionis Pharmaceuticals, Inc.
For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has six marketed medicines and a leading pipeline in neurology, cardiology, and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter), LinkedIn and Instagram.
Ionis Forward-looking Statements
This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of our commercial medicines, eplontersen, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions, or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended Dec. 31, 2024, and most recent Form 10-Q, which are on file with the SEC. Copies of these and other documents are available at www.Ionis.com.
Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc. WAINUA™ is a trademark of AstraZeneca plc.
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Source: Ionis Pharmaceuticals, Inc.
FAQ
What are the key benefits shown in WAINZUA's Phase 3 trial for ATTRv-PN treatment?
How is WAINZUA administered to ATTRv-PN patients?
Which regions have approved IONS's WAINZUA/WAINUA for ATTRv-PN treatment?
What is the partnership structure between IONS and AstraZeneca for WAINZUA?
