WAINZUA (Eplontersen) Receives EU Approval for Hereditary ATTR Amyloidosis Treatment
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that WAINZUA (eplontersen), developed in collaboration with AstraZeneca, has been approved by the European Union (EU) for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) in adult patients with stage 1 or stage 2 polyneuropathy. This approval marks a significant milestone for patients suffering from this debilitating condition, providing them with a novel, self-administered treatment option.
A Groundbreaking Treatment Option for ATTRv-PN Patients
WAINZUA is now the only approved therapy in the EU that can be self-administered on a once-monthly basis via an auto-injector. This innovation enhances patient convenience and accessibility, allowing for a more manageable treatment regimen compared to other available therapies that require more frequent or clinical administration.
The European Commission (EC) granted the approval following the positive recommendation from the Committee for Medicinal Products for Human Use (CHMP), a key regulatory body responsible for evaluating medicinal products in the EU. The decision was primarily based on the compelling results from the NEURO-TTRansform Phase 3 trial, which demonstrated WAINZUA’s efficacy in treating patients with ATTRv-PN.
NEURO-TTRansform Phase 3 Trial: A Strong Basis for Approval
The NEURO-TTRansform Phase 3 trial was a multicenter, randomized, placebo-controlled study designed to evaluate the safety and efficacy of WAINZUA in patients diagnosed with hereditary transthyretin-mediated amyloidosis with polyneuropathy. The study included patients in various geographical regions and followed them for a period of 66 weeks to assess long-term efficacy and safety.
The results demonstrated that WAINZUA provided consistent and sustained benefits in treating ATTRv-PN. The trial met its co-primary endpoints, which included:
- Reduction in Serum Transthyretin (TTR) Concentration: Patients who received WAINZUA showed a significant decrease in TTR protein levels compared to baseline, indicating effective suppression of disease progression.
- Neuropathy Impairment Measured by mNIS+7: The modified Neuropathy Impairment Score +7 (mNIS+7) was used to assess neuropathy progression, and WAINZUA-treated patients experienced a marked improvement compared to an external placebo control group.
Additionally, the study assessed secondary endpoints, including:
- Quality of Life (QoL) Improvements: Patients taking WAINZUA reported better quality of life, as measured by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN). This improvement underscores the meaningful clinical impact of WAINZUA in managing symptoms and enhancing daily living for those with ATTRv-PN.
Throughout the duration of the trial, WAINZUA maintained a favorable safety and tolerability profile, reinforcing its potential as a well-tolerated therapeutic option for patients battling ATTRv-PN.
Understanding Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy (ATTRv-PN)
ATTRv-PN is a rare, progressive, and life-threatening disease caused by the accumulation of misfolded transthyretin (TTR) protein in peripheral nerves and various organs. This buildup results in nerve damage, leading to symptoms such as loss of motor function, sensory impairment, autonomic dysfunction, and ultimately severe disability. Without treatment, most patients experience significant motor impairment within five years of diagnosis, and the disease can prove fatal within a decade.
WAINZUA is a once-monthly RNA-targeted therapy designed to specifically reduce the production of TTR protein at its source, thereby slowing disease progression and improving patient outcomes. By lowering TTR protein levels, WAINZUA effectively addresses the underlying pathology of ATTRv-PN rather than merely managing symptoms, making it a transformative option for affected individuals.
A Global Effort: Expanding Patient Access
The approval of WAINZUA in Europe follows previous regulatory successes in North America and other key regions worldwide. The drug was initially approved under the brand name WAINUA in the United States in December 2023 for the treatment of ATTRv-PN and has since received approvals in Canada and the United Kingdom. These approvals underscore the therapy’s clinical value and highlight the growing global recognition of its potential benefits.
Brett P. Monia, Ph.D., Chief Executive Officer of Ionis Pharmaceuticals, emphasized the significance of this milestone:
“Today’s approval of WAINZUA in Europe offers adults with hereditary transthyretin-mediated amyloidosis with polyneuropathy a new, self-administered treatment option that provides consistent suppression of transthyretin production and improves neuropathy impairment and quality of life. With approvals in North America, the UK, and now across the EU, we are proud of the continued progress as we and our partner, AstraZeneca, rapidly and effectively deliver WAINZUA to people around the world.”
Collaboration Between Ionis and AstraZeneca
The development and commercialization of WAINZUA are the result of a strategic collaboration between Ionis Pharmaceuticals and AstraZeneca. Under the terms of their global agreement, the two companies are jointly responsible for developing and marketing WAINUA in the United States. Meanwhile, AstraZeneca holds exclusive commercialization and development rights in all other regions worldwide.
This partnership enables a streamlined approach to bringing WAINZUA to market in multiple regions, ensuring that patients in need have access to this groundbreaking therapy as swiftly as possible. AstraZeneca’s global reach and expertise in commercializing innovative therapies further bolster the potential success of WAINZUA on an international scale.
Future Outlook and Ongoing Regulatory Submissions
Following this EU approval, AstraZeneca and Ionis plan to continue their efforts to secure regulatory approvals in additional regions where unmet medical needs for ATTRv-PN persist. The companies have already initiated applications for market authorization in several other countries, with regulatory reviews ongoing.
Moreover, research and development efforts remain a priority, as Ionis and AstraZeneca explore additional potential applications for WAINZUA beyond ATTRv-PN. Given the drug’s ability to reduce TTR protein levels effectively, it may have therapeutic implications for other forms of transthyretin-mediated amyloidosis (ATTR), including cardiomyopathy-associated variants (ATTR-CM), which affect the heart.
